NeurologyLive® Mind Moments®

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, epilepsy expert Imad Najm, MD, PhD, sat down to discuss the upcoming Cleveland Clinic Epilepsy Summit, an educational symposium that takes place September 11-15, in Cleveland, Ohio. Najm, who serves as the director of Cleveland Clinic's Epilepsy Center at the Cleveland Clinic Neurological Institute, spoke about the objectives and goals of the meeting, including updates on sEEG, epilepsy surgery, treatments for drug-resistant epilepsy, and gene therapy. In addition, he provided an overview of the latest advances in epilepsy surgery, genetics, and seizure controlling approaches. Furthermore, he spoke on some of the educational challenges with adjusting to changes in neurosurgery, the potential benefits and limitations of gene therapy, and the future of epilepsy treatment. Looking for more Epilepsy discussion? Check out the NeurologyLive® Epilepsy clinical focus page.Episode Breakdown: 1:10 – Mission and goals of Epilepsy Summit 3:05 – Overviewing sessions and educational attainment 4:55 – Changes to epilepsy surgery, advances over the years 9:15 – Neurology News Minute 11:50 – Learning curve for understanding new epilepsy surgery tactics 13:50 – Closing thoughts on the upcoming meeting 15:50 – Potential benefits and challenges to gene therapy for epilepsy The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: EMA Accepts Marketing Authorization Application for Huntington Disease Agent Pridopidine BTK Inhibitor Tolebrutinib Slows Disability Progression in Phase 3 HERCULES Study of Non-Relapsing Secondary Progressive MS FDA Accepts Resubmitted New Drug Application for Migraine Treatment AXS-07 Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Sharon Cohen, MD, a behavioral neurologist, sat down to discuss positive data from an ongoing phase 1 study of mivelsiran (Alnylam Pharmaceuticals), the first investigational RNA interference therapeutic targeting amyloid precursor protein for Alzheimer disease (AD). Cohen, who also serves as the medical director of the Toronto Memory Program at the University of Toronto, discussed the potential of RNA therapeutics for treating AD, the unique mechanism of action of mivelsiran, and some of the early promising safety, efficacy, and pharmacokinetic data observed in the phase 1 trial. In addition, Cohen touched upon the idea of how RNA therapeutics could be used in combination with previously approved novel treatments and the benefits mivelsiran brings with no observed amyloid-related imaging abnormalities. Furthermore, the discussion covered some of the potential of this investigational agent, what to expect in the multi-dose part B of the study, and an additional phase 2 study in cerebral amyloid angiopathy. Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page.Episode Breakdown: 1:15 – Mechanism of action of mivelsiran and how it can be potentially beneficial in AD 4:15 – Growing knowledge of RNA therapeutics and their impact on neurologic conditions 7:00 – Neurology News Minute 10:10 – Phase 1 study data, including efficacy, safety, biomarker, and pharmacokinetic results 19:20 – How mivelsiran fits with other approved therapies for AD; future goals and directions of the drug The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Clears Indapta Therapeutics’ IND for Cell Therapy IDP-023 in Progressive Multiple Sclerosis Muscle-Targeting Therapy Apitegromab Effective in Spinal Muscular Atrophy Over 4 Year Period FDA Approves Medtronic’s Deep Brain Stimulation Technology for Asleep Capabilities Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Phil Jochelson, therapeutic head for Clinical Development Neuroscience at Jazz Pharmaceuticals, sat down to discuss the phase 4 XYLO study, a randomized controlled study evaluating the effects of switching from high-sodium to low-sodium oxybate on blood pressure in patients with narcolepsy. Jochelson gave some insight regarding the conduct of the trial, including end points, objectives, and patient demographics, as well as some of the benefits to studying blood pressure and cardiovascular risks. In addition, he discussed enhancements to narcolepsy care through low-sodium oxybate, the importance of unique studies like XYLO, and the need to educate clinicians on sodium load and its risks. Looking for more sleep disorders discussion? Check out the NeurologyLive® sleep disorders clinical focus page. Episode Breakdown: 1:15 – Reasons behind the XYLO study and links between high sodium intake and cardiovascular risk 4:45 – Patient demographics; inclusion and exclusion criterias 6:40 – Neurology News Minute 9:25 – Overview of XYLO and determining what a successful study looks like 15:30 – Improved understanding of the benefits of low-sodium oxybate The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: EU Regulatory Review Adopts Negative Opinion of Lecanemab as Treatment for Alzheimer Disease GLP-1 Agonist Liraglutide Shows Protective Effects on Alzheimer Disease in Phase 2 Trial FDA Approves Carbidopa/Levodopa ER Capsules Formulation IPX203 for Parkinson Disease Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Michael Weiss, chief executive officer of TG Therapeutics, discussed ublituximab (Briumvi), a recently approved anti-CD20 therapy for relapsing multiple sclerosis, and the advantages it brings to patients. Specifically, he talked about the Department of Veteran Affairs' decision to award national contract to ublituximab as the preferred anti-CD20 for this patient population, and how this facilitates greater access to the treatment. In addition, he discussed how the knowledge profile of the agent has grown over time, as well as the additional efforts to gain a better understanding of its efficacy and safety. Furthermore, he spoke on the differences between ublituximab and other approved agents, and why it may be more applicable to certain patients. Looking for more multiple sclerosis discussion? Check out the NeurologyLive® Multiple sclerosis clinical focus page.Episode Breakdown: 1:05 – Significance of ublituximab awarded national contract as preferred anti-CD20 for relapsing MS 3:30 – Advantages and differences of ublituximab vs approved therapies for MS 11:40 – Neurology News Minute 14:30 – Growing knowledge profile of ublituximab over the years 19:05 – Future plans of ubtlituximab; long-term goals of the therapy The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Eli Lilly Announces Date for Donanemab FDA AdComm Hearing Patient Death Reported in Phase 2 DAYLIGHT Study of Pfizer’s Gene Therapy for Duchenne ALS Candidate PrimeC Shows Greater Treatment Effect in High-Risk ALS Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Douglas Wajda, PhD, an assistant professor of neurology at Cleveland State University, discussed a pilot study presented at the 2024 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting using a functional electrical stimulation approach, Neural Sleeve, to treat gait in MS. Wajda, who has been studying gait in MS for several years, gave clinical insight on the functionality and feasibility of this approach, and the personalized treatment patients can gain from it. He also discussed the pilot study in detail, including the major end points and takeaways from the small group of patients observed. Looking for more multiple sclerosis discussion? Check out the NeurologyLive® multiple sclerosis clinical focus page.Episode Breakdown: 1:00 – Functionality of Neural Sleeve 2:45 – Feasibility and self-administration of Neural Sleeve 3:50 – Neurology News Minute 5:50 – Greatest clinical takeaways from pilot study 8:15 – Steps in advancing this therapeutic approach 9:45 – Challenges with gait in MS and personalizing treatment methods The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Gene Therapy LX2006 Positively Impacts Cardiac Biomarkers of Friedreich Ataxia, Clinical Data Show Buntanetap Transitioning to Crystal Form Following FDA Go-Ahead Endo Voluntarily Recalls One Lot of Clonazepam Tablets Following Mislabeling Error Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Sameea Husain-Wilson, DO, a movement disorder specialist at the Marcus Neuroscience Institute of Baptist Health, provided clinical perspective on the use of the Syn-One diagnostic test for patients with Parkinson disease (PD). She gave an overview of how the test is utilized in clinic, ways to interpret results, and the right personnel needed to ensure an accurate diagnosis. In addition, she provided clarity on the role of alpha-synuclein in PD, the advances in research in this area, and how the test incorporates this prominent biomarker. Furthermore, Husain-Wilson detailed some of the next steps in further optimizing the test in clinical settings, highlighting the importance of other non-movement disorder specialists who will play a major role in the diagnostic evaluation of future patients with PD.Looking for more movement disorder discussion? Check out the NeurologyLive® movement disorder clinical focus page.Episode Breakdown: 1:10 – Step by step process on how Syn-One test is used; considerations after process is done 3:15 – How the test differentiates PD from other neurodegenerative disorders 9:50 – Neurology News Minute 12:20 – Value and role in assessing alpha-synuclein in PD 14:50 – Ways to further optimize Syn-One going forward The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Eli Lilly's Donanemab for Early Symptomatic Alzheimer Disease Buntanetap Improves Motor, Nonmotor and Cognitive Symptoms of Parkinson Disease in Phase 3 Study Gene Therapy AMT-130 Slows Huntington Disease Progression in Interim Phase 1/2 Trials Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question.In this episode, we're covering the recent approval of donanemab as a new treatment for adults with early symptomatic Alzheimer disease (AD). Marketed as Kisunla, donanemab's approval marks the third antiamyloid therapy to get FDA greenlight for early-stage AD, following the controversial approval of aducanumab (Aduhelm; Biogen) in 2021 and lecanemab (Leqembi; Eisai) in 2023. Donanemab, administered as a 350 mg/20 mL once-monthly injection for intravenous infusion, had its approval supported by the phase 3 TRAILBLAZER-ALZ-2 trial (NCT04437511), a large-scale, double-blind, placebo-controlled trial that featured 1736 patients with early-stage AD. Following the approval, NeurologyLive sat down with Joel Salinas, MD, MBA, a behavioral neurologist at NYU Langone and clinical assistant professor in the department of neurology at the NYU Grossman School of Medicine. Salinas, who also serves as the chief medical officer at Isaac Health, discussed the positive impacts of the approval, the importance of patient selection for the medication, and how clinicians should discuss its benefits and harms to patients. In addition, he commented on how approvals like donanemab continue to carry momentum in the AD field going forward. For more of NeurologyLive's coverage of donanemab's approval, head here: FDA Approves Eli Lilly's Donanemab for Early Symptomatic Alzheimer Disease Episode Breakdown: 2:10 – Positive downstream impacts of donanemab's approval 4:20 – Considerations and caution with prescribing donanemab 6:05 – Salinas on patient-clinician conversations about AD treatments 8:00 – Closing remarks and continued progress in AD field Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Anne Marie Morse, DO, FAAN, a pediatric neurologist and sleep medicine specialist at Geisinger Medical Center, sat down at the 2024 SLEEP Annual Meeting to discuss a poster presentation highlighting a listening analysis that characterized the struggles and unmet needs of people with narcolepsy. in the discussion, Morse outlined some of the major findings, including the rates of missed and misdiagnosis, how cataplexy impacts daily life, and some of the other comorbidities associated with the condition. She spoke on the value of gaining patient perspectives and using their descriptions for their disorders helps in understanding true unmet needs. Furthermore, she spoke on the importance of a strong patient-clinician relationship, the ability to communicate openly, and the steps following diagnosis to ensure effective long-term treatment outcomes. Looking for more sleep disorders discussion? Check out the NeurologyLive® sleep disorders clinical focus page.Episode Breakdown: 1:10 – Motivations behind conducting listening analysis 3:15 – Major clinical takeaways from study; notable quality of life impairments in narcolepsy 7:00 – Impact of cataplexy and patients' overreaction 9:50 – Neurology News Minute 12:20 – Reasons for missed and misdiagnosis 14:50 – Early tips to pathing patients towards treatment success The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Grants Traditional Approval to Elevidys Gene Therapy for Ambulatory DMD, Accelerated Approval for Nonambulatory Patients FDA Approves Efgartigimod as New Treatment for Chronic Inflammatory Demyelinating Polyneuropathy FDA Approves Pitolisant for Excessive Daytime Sleepiness in Pediatric Narcolepsy Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, we spoke with a variety of specialists in neurology who presented research and gave talks at the 2024 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting, held May 29-June 2, in Nashville, Tennessee. Those included in this week's episode, in order of appearance, are: Anthony Feinstein, PhD, FRCPC, MBBCh, a professor of psychiatry at the University of Toronto. Brian G. Weinshenker, MD, a professor of neurology at the University of Virginia. Douglas A. Wajda, PhD, an assistant professor of neurology and member of the Health and Human Performance Department in the College of Education at Cleveland State University. Le Hua, MD, director of Clinical Operations and director of the Multiple Sclerosis Program at Cleveland Clinic's Lou Ruvo Center for Brain Health in Las Vegas, Nevada. Eion P. Flanagan, MB, BCh, a professor of neurology and chief of the Division of Multiple Sclerosis and Autoimmune Neurology at Mayo Clinic; and director of the Autoimmune Neurology Fellowship. Want more from the 2024 CMSC Annual Meeting? Click here for all of NeurologyLive®'s coverage of CMSC 2024.Episode Breakdown: 1:40 – Feinstein on the difficulties with improving fatigue in multiple sclerosis and the lack of improvement seen from approved disease-modifying therapies. 5:20 – Weinshenker on the key diagnostic aspects of neuromyelitis optica spectrum disorder and how it differs from other similarly presenting autoimmune disorders. 9:40 – Wajda on the use of the Cionic Neural Sleeve, the advantages it holds, and how it may be used to treat gait dysfunction in multiple sclerosis. 12:00 – Hua on the differential diagnosis of pediatric-onset MS vs late-onset MS, and the notable biologic changes observed. 19:30 – Flanagan on the diagnostic pearls for MOG-antibody associated disease and the importance of early recognition of this group. Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Katherine Peters, PhD, a neurologist and neurooncologist at the Preston Robert Tisch Brain Tumor Center at Duke University provided context on a new analysis from the phase 3 INDIGO trial, a study assessing vorasidenib in patients with mutant isocitrate dehydrogenase (mIDH) 1/2 diffuse gliomas. The conversation, which occurred at the 2024 AAN Annual Meeting, covered the covered the therapeutic potential of this agent and its impacts on quality of life, neurocognition, and seizure control. Peters, an expert in the field, provided insight on the mechanism of action of vorasidenib, the added value behind the exploratory analysis, and the next steps in research. Furthermore, she provided context on the patient sample observed and why these data may hold significant weight going forward. Looking for more neuromuscular discussion? Check out the NeurologyLive® epilepsy clinical focus page.Episode Breakdown: 1:05 – Mechanism of action of vorasidenib, a mIDH 1/2 inhibitor 2:00 – Overview of exploratory analysis results 4:20 – Significance of new data, how it adds to previous primary and secondary outcomes 5:50 – Neurology News Minute 8:25 – Next steps for the study and use of vorasidenib 10:35 – Remaining unmet needs for patients with diffuse gliomas The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Diazepam Buccal Film’s Role in Treating Intermittent Pediatric Seizures, with Michael Rogawski, MD, PhD Ocrelizumab Gains EU CHMP Positive Opinion for Subcutaneous Formulation to Treat MS FDA Approves Sprinkle Formulation of Neurocrine Biosciences’ Valbenazine for Tardive Dyskinesia or Huntington disease Chorea Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Justin Klee and Josh Cohen, cofounders and cochief executive officers of Amylyx Pharmaceuticals, sat down at the recently concluded AAN Annual Meeting to discuss the results from the pivotal phase 3 PHOENIX trial (NCT) of AMX0035 (Relyvrio) in patients with amyotrophic lateral sclerosis (ALS). The duo provided clarity on the findings, positive takeaways from the disappointing data, and the lessons learned in ALS drug development. In addition, the two provided commentary on the future plans of the therapy in other tauopathies like progressive supranuclear palsy and neurologic conditions like Wolfram syndrome. Furthermore, Klee and Cohen shared thoughts on the company's drug pipeline, including AMX0114, an antisense oligonucleotide in development for patients with ALS. Looking for more neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page. Episode Breakdown: 1:05 – Decision behind removing AMX0035 from market 3:00 – Lessons from PHOENIX, AMX0035 drug development program 6:45 – Unraveling PHOENIX study data 10:00 – Neurology News Minute 12:10 – Potential of AMX0035 in other tauopathies, neurologic conditions 15:00 – Outlook of antisense oligonucleotide AMX0114 in ALS The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Diazepam Buccal Film’s Role in Treating Intermittent Pediatric Seizures, with Michael Rogawski, MD, PhD Ocrelizumab Gains EU CHMP Positive Opinion for Subcutaneous Formulation to Treat MS FDA Approves Sprinkle Formulation of Neurocrine Biosciences’ Valbenazine for Tardive Dyskinesia or Huntington disease Chorea Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of diazepam buccal film (Libervant; Aquestive Therapeutics) for the treatment of pediatric patients with intermittent, stereotypic episodes of frequent seizure activity (seizure clusters, acute repetitive seizures). Indicated for those between 2 and 5 years of age, the therapy offers patients a compact, easily administered diazepam formulation. The FDA previously granted tentative approval in August 2022 for Libervant for treatment of these patients with epilepsy 12 years of age and older, with U.S. market access for Libervant for this age group of patients subject to the expiration of the existing orphan drug market exclusivity of a previously FDA approved drug scheduled to expire in January 2027. Following the approval, Michael Rogawski, MD, PhD, a distinguished professor of neurology and pharmacology at the University of California Davis Health Medical Center, provided insight on what the decision means for patients and clinicians. Rogawski gave comment on the significance of having a new administration route for diazepam, the safety and feasibility of diazepam buccal film, and some of the major points of emphasis from its clinical program. For more of NeurologyLive's and Contemporary Pediatrics coverage of diazepam buccal film's approval, head here: FDA approves diazepam for seizure clusters in patients 2 to 5 years Episode Breakdown: 0:20 – Diazepam buccal film approved for intermittent seizures in pediatrics 2:00 – Michael Rogawski, MD, PhD, giving reaction to the approval 4:10 – Flexibility with multiple diazepam administration routes 6:55 – Rogawski on the safety of diazepam buccal film amid high need 12:30 – Notable takeaways from the diazepam buccal film trial program Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Ian Kremer, executive director of the leaders Engaged on Alzheimer's Disease (LEAD) coalition, spoke about several relevant topics regarding Alzheimer care as new novel therapeutics emerge. He talked about the lessons learned from the recent discontinuation of aducanumab, and the potential and limitations lecanemab (Leqembi; Eisai) and donanemab (Eli Lilly) may bring. Additionally, he provided comments about what matters to patients, the perception of the FDA approval process, and what is considered "clinically meaningful." Furthermore, he gave perspective on ways to improve drug development and emphasized the need for policy decisions to be based on scientific evidence and not by sensationalized headlines. Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page. Episode Breakdown: 1:10 – Promise in the Alzheimer field in 2024 4:05 – Ways of improving efficiencies with drug develpment 9:10 – Discontinuation of aducanumab 11:10– Neurology News Minute 13:50 – Lessons learned from aducanumab, antiamyloid therapies 22:10– Conversations between clinicians and patients surrounding expectations/limitations of antiamyloid therapies and available treatments The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Agrees to New Specialized Protocol for Phase 3b Study of ALS Agent NurOwn FDA Approves Alternate Administration Routes for Antiseizure Medication Cenobamate Extended Use of Investigational Agent IPX203 Safe in Parkinson Disease Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Francesco Saccà, MD, PhD, an associate professor of neurology at the University of Naples, discussed an ongoing study assessing the use of dimethyl fumarate, an approved therapy for relapsing multiple sclerosis, in patients with Friedreich ataxia (FA). He spoke on the mechanism of action of the agent and why it can be beneficial in this patient population, as well as how it differs from omaveloxolone (Skyclarys; Biogen), the first approved treatment for FA. In addition, he outlined the study design, the primary end point of frataxin decrease, and what would be considered a "successful" study. Furthermore, he spoke on the reasons behind the trial and what led investigators to this point. Looking for more ataxia discussion? Check out the NeurologyLive® ataxia clinical focus page. Episode Breakdown: 1:05 – Origin of the study 3:00 – Conduct of the study, end points, goals 5:10 – Promising mechanism of action of dimethyl fumarate to treat Friedreich ataxia 7:30 – Questions in clinical impact of changes in frataxin 10:00 – Neurology News Minute 12:40 – What is considered a successful study? 14:55 – Patient inclusion, demographic makeup of the study 16:35 – Timeline of the trial and data readout The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Alexion's Ravulizumab-cwvz for Neuromyelitis Optica Spectrum Disorder Epilepsy Agent STK-001 Demonstrates Disease-Modifying Effects in Early Phase Studies of Dravet Syndrome Eisai Submits sBLA for Monthly Intravenous Maintenance Dosing of Alzheimer Therapy Lecanemab Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of givinostat (Duvyzat; Italfarmaco) for the treatment of Duchenne muscular dystrophy (DMD). The therapy, a proprietary histone deacetylase (HDAC) inhibitor, was approved as the first nonsteroidal drug for patients with all genetic variants of DMD. The supporting data for the approval of givinostat comes from the phase 3 EPIDYS trial (NCT02851797). EPIDYS, a randomized, double-blind, placebo-controlled, multicenter study, included 179 ambulant male individuals who were randomly assigned 2:1 to either oral givinostat or placebo for an 18-month treatment period. Following the approval, Sharon Hesterlee, PhD, executive vice president and chief research officer of the Muscular Dystrophy Association, sat down to discuss the significance of the approval and how it changes the care for patients with DMD. She spoke specifically about the mechanism of action of the therapy, its safety profile, and how it may be used with other agents. In addition, she discussed other related topics on gene therapy and unmet needs for this patient population. For more of NeurologyLive's coverage of givinostat's approval, head here: FDA Approves Italfarmaco's Givinostat for Duchenne Muscular Dystrophy Episode Breakdown: 0:30 – Givinostat approved for Duchenne muscular dystrophy 2:00 – Sharon Hesterlee, PhD, on the approval's implications 3:35 – Positive downstream effects of the approval 4:50 – Hersterlee on the safety profile of givinostat 5:40 – Promising outlook of the DMD field 6:45 – Overcoming roadblocks involved with gene therapy 8:55 – Remaining unmet needs for patients with Duchenne 10:15 – Closing thoughts on the approval Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Daniel Ontaneda, MD, an associate professor of neurology at the Cleveland Clinic Lerner College of Medicine, sat down to discuss the recently concluded Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum. Ontaneda, program committee vice-chair of the meeting, provided insight on a number of notable sessions and discussions that highlighted the recent advances in the MS field. In addition, he talked about the need to innovate clinical trials, ways to tackle progressive MS, and whether drug trials are truly assessing patients at the right time points. Furthermore, he discussed multi-stage trials, the potential role of Bruton tyrosine kinase (BTK) inhibitors, and how this year's meeting differs from previous ones. Looking for more multiple sclerosis discussion? Check out the NeurologyLive® multiple sclerosis clinical focus page. Episode Breakdown: 1:05 – Overviewing sessions from ACTRIMS Forum 13:10 – Innovating clinical trial design 16:15 – 2023 ACTRIMS vs 2024 ACTRIMS 20:20 – Neurology News Minute 23:30 – Testing combination approaches for progressive MS 26:45 – The impact of timing therapeutic interventions This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA AdComm to Review Investigational Alzheimer Agent Donanemab and Phase 3 Results Amylyx Mulls Over Pulling AMX0035 Following Disappointing Phase 3 PHOENIX Findings FDA Issues Complete Response Letter for Long-Acting Form of Glatiramer Acetate for Relapsing Multiple Sclerosis Phase 2 PROCEED Trial of PACAP-Targeting Antibody Lu AG09222 in Migraine Announced Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Alcibiades Rodriguez, MD, medical director of the Comprehensive Epilepsy Center—Sleep Center at NYU Langone discussed several sleep-related topics, including the diagnosis of rare sleep disorders and hypersomnias. He spoke on the treatment of obstructive sleep apnea, the importance of adherence to medication, and the ways clinicians can help patients stick to their treatment regimen. Furthermore, he talked about the conversations between patients and clinicians to ensure an accurate diagnosis, as well as the emerging research in the sleep disorder field over the coming years.Looking for more sleep disorders discussion? Check out the NeurologyLive® sleep disorders clinical focus page.Episode Breakdown: 1:40 – Conversations needed to differentiate diagnoses 4:30 – Appearance of sleep disorders and overlap 6:50 – Complications with untreated sleep apnea 8:25 – Neurology News Minute 10:55 – Ways to improve adherence to medication 13:50 – Emerging research in the field This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Glatiramer Acetate Depot Demonstrates Sustained Longterm Safety Profile as Potential MS Therapy Phase 3 DAYBREAK Trial Highlights Long-Term Efficacy of Ozanimod for Relapsing Multiple Sclerosis SRP-9001 Improves Duchenne Muscular Dystrophy Disease Trajectory Despite Failing to Meet Primary End Point in Phase 3 EMBARK Trial Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Stephen From, chief executive officer of Aruna Bio, sat down to discuss the company's investigational exosome candidate AB126 and its upcoming phase 1b/2a trial in patients with ischemic stroke. He discussed the mechanism of action of the therapy, the advantages of its anti-inflammatory and neuroprotective properties, and how it will be used with other stroke treatments. Furthermore, he gave insight on the complexities with conducting stroke trials and improving neurodegeneration in poststroke patients. Looking for more stroke discussion? Check out the NeurologyLive® stroke clinical focus page. Episode Breakdown: 1:00 – Overviewing phase 1b/2a trial 7:05 – Mechanism of action of AB126 11:15 – Neurology News Minute 13:45 – Role of AB126 in stroke treatment landscape 17:50 – Challenges with repairing neurodegeneration in stroke This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: European Commission Approves Omaveloxolone as First Therapy for Friedreich Ataxia AVP-786 Falls Short in Phase 3 Study of Alzheimer Disease Agitation FDA Grants Priority Review to Efficacy Supplement for Sarepta Therapeutics’ SRP-9001 Indication Expansion Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Ignacio Mata, PhD, sat down to discuss a recently published meta-analysis on the genetic backgrounds of Parkinson disease beyond traditional European populations. Mata, an associate professor in the Genomic Medicine Institute at Cleveland Clinic, provided insight on the newly identified novel loci and how the data adds to previous genome-wide association studies. In addition, he gave thoughts on the possibility of gene therapy and some of the barriers that come with it, as well as the feasibility of incorporating genetic checkpoints into clinical trial inclusion criteria. Looking for more movement disorder discussion? Check out the NeurologyLive® movement disorder clinical focus page. Episode Breakdown: 1:10 – Meta-analysis overview 3:20 – Significance of results, how they add to the literature 6:50 – Feasibility of incorporating genetic screening into clinical trials 8:40 – Neurology News Minute 11:15 – Realistic expectations and potential with gene therapy 15:45 – Next steps in advancing this research This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Biogen Walks Away From Aducanumab to Prioritize Lecanemab and Alzheimer Disease Pipeline Updated Guidelines Published to Improve Care in Specialized Epilepsy Centers FDA Clears IND for Gene Therapy Candidate ETX101 in Dravet Syndrome Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Orrin Devinsky, MD, director of the NYU Langone Comprehensive Epilepsy Center, provided commentary on a recently published study assessing video evidence of sudden unexplained deaths in toddlers. Devinsky, who also serves as a professor of neurology, discussed the importance behind crib cams, the moments captured prior to the deaths assessed, and the involvement of seizure activity even with no prior history. In addition, he spoke on the abnormal sounds and movements of these children, the ways to advance SUDC research, and the next steps in understanding and relaying the data. Looking for more epilepsy discussion? Check out the NeurologyLive® epilepsy clinical focus page.Episode Breakdown: 1:10 – Reasons behind studying sudden death in toddlers 3:55 – Notable takeaways from trial results 8:15 – Advancing ways to research SUDC/SUDEP 9:40 – Neurology News Minute 12:05 – Realistic ways to lower convulsive activity prior to sleep 15:05 – What abnormal movements/noises can ellucidate 15:55 – Ways to expand the research further This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Takeda’s Immune Globulin Infusion, Hyqvia, for Chronic Inflammatory Demyelinating Polyneuropathy FDA Issues Complete Response Letter for Satsuma’s DHE Nasal Powder STS101 to Treat Acute Migraine ALS Agent PrimeC to Advance to Phase 3 Study After Positive PARADIGM Trial Data Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Cathleen Lutz, PhD, vice president of the Rare Disease Translational Center at the Jackson Laboratory, provided comment on a recently published study which unveiled that stathmin-2 loss leads to neurofilament-dependent axonal collapse in patients with amyotrophic lateral sclerosis (ALS). Lutz offered insight on the role stathmin-2 may have in this disease, the questions that still remain, and whether restoration of stathmin-2 may be an attractive therapeutic approach. Furthermore, she spoke on the potential of this protein as a disease-modifying biomarker and whether it may be incorporated in trials in the near future. Looking for more neuromuscular discussion? Check out the NeurologyLive® neuromuscular clinical focus page.Episode Breakdown: 1:15 – Idea behind studying stathmin-2 2:45 – Overviewing study results 7:05 – Next steps in extending this research 8:40 – Neurology News Minute 11:30 – Stathmin-2's connection with neurofilament light 12:55 – Potential of stathmin-2 as ALS-specific biomarker 14:45 – Future use of stathmin-2 in clinical trials 15:30 – Closing thoughts This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Clears Phase 2 KYSA-7 Study of CAR T-Cell Therapy in Refractory Progressive Multiple Sclerosis Dyne Reports Positive Phase 1/2 Data for Duchenne Agent DYNE-251 Parkinson Gene Therapy AB-1005 Meets Primary End Point in Phase 1b Trial VISIONARY-MS Trial Reports CNM-Au8 Improves Vision and Cognition in Multiple Sclerosis Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, we comprised some of the most interesting conversations from Mind Moments podcast episodes in 2023, highlighting research in neurology that moves the field forward. The NeurologyLive team included several reaction episodes to FDA approved medicines, providing clinicians an overview of what they can expect from these new therapies and how they will be used in clinic going forward. Those included in this week's episode, in order of appearance, are: Sharon Cohen, MD, FRCPC, behavioral neurologist and medical director of the Toronto Memory Program at the University of Toronto, and a trial investigator for Clarity AD, the phase 3 trial of lecanemab. Check out the full episode here: https://www.neurologylive.com/view/special-episode-lecanemab-approved-for-alzheimer-disease Robert A. Hauser, MD, MBA, director of the Parkinson’s and Movement Disorders Center at the University of South Florida.Check out the full episode here: https://www.neurologylive.com/view/episode-81-widening-therapeutic-window-parkinson-disease Alberto Espay, MD, PhD, the division director and Research Endowed Chair of the James J. and Joan A. Gardner Family Center for Parkinson’s Disease and Movement Disorders, and a professor of clinical neurology and rehabilitation medicine at the University of Cincinnati.Check out the full episode here: https://www.neurologylive.com/view/episode-94-discussion-leqembi-lecanemab-approval-alzheimer-disease Carolina Ferreira Atuesta, MD, MSc, a scientific researcher and clinical data manager at the Icahn School of Medicine at Mount Sinai.Check out the full episode here: https://www.neurologylive.com/view/episode-82-managing-seizures-after-ischemic-stroke Natalie Goedeker, CPNP, a nurse practitioner in neurology in the Neuromuscular Division at Washington University in St Louis.Check out the full episode here: https://www.neurologylive.com/view/special-episode-tofersen-approved-sod1-als Want more Mind Moments episodes? Click here for all of NeurologyLive®'s podcast episodes.Episode Breakdown: 1:20 – Cohen on the safety of lecanemab, including the 3 patient deaths reported in the supportive clinical trial program. 6:25 – Hauser on the clinical advantages of IPX203, a potential new carbidopa/levodopa formulation for patients with Parkinson disease. 9:20 – Espay on tempering expectations of lecanemab and other antiamyloid therapies in the treatment of Alzheimer disease. 13:20 – Atuesta on the lack of standardized methods for monitoring patients with poststroke seizures, including elders. 17:00 – Goedeker on the approval of gene therapy SRP-9001 for patients with Duchenne muscular dystrophy, the integration process, and safety considerations clinicians should be aware of. This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Jennifer Gudeman, senior vice president of medical and clinical affairs for Avadel Pharmaceuticals, sat down to discuss a recently published post-hoc analysis of the phase 3 REST-ON trial of once-nightly sodium oxybate (Lumryz). In the conversation, Gudeman talked about the impact of the drug on both narcolepsy types 1 and 2, the differences between these 2 subtypes, and the ways clinical trials of narcolepsy agents continue to evolve. Furthermore, she spoke on some of the unmet needs for this patient population, including lingering brain fog and overcoming negative stigmas. Looking for more sleep disorders discussion? Check out the NeurologyLive® narcolepsy clinical focus page. Episode Breakdown: 1:10 – Overview of post-hoc analysis of REST-ON 3:15 – Differences in type 1 and 2 narcolepsy 5:50 – Neurology News Minute 8:15 – Expanding treatment profile of once-nightly sodium oxybate 10:55 – Lingering unmet needs of patients with narcolepsy 11:20 – Realistic timeline of gene therapies for epilepsy 14:00 – Future of narcolepsy research, drug development trials This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Places Clinical Hold on Roche’s BTK Inhibitor Fenebrutinib for Multiple Sclerosis ALS Candidate PrimeC Meets Primary Safety and Secondary End Points in Phase 2b PARADIGM Study FDA Clears NeuroOne’s OneRF Ablation System for Neurosurgerical Procedures Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, William Gallentine, DO, interim chief of pediatric neurology at Stanford Medicine Children’s Health, who discussed several topics related to the advances in pediatric epilepsy, epilepsy surgery, and seizure localization. Gallentine, who also serves as a clinical professor, provided insight on the screening process and eligibility criteria for epilepsy surgery, as well as the importance of lifestyle modifications when treating epilepsy. Furthermore, he touched upon the genetic background of certain epileptic disorders and when the clinical community can expect gene therapies. Looking for more epilepsy discussion? Check out the NeurologyLive® epilepsy clinical focus page. Episode Breakdown: 1:10 – State of pediatric epilepsy 2:15 – Screening potential candidates for epilepsy surgery 5:00 – Advances in seizure localization 7:15 – Neurology News Minute 9:25 – Balance of nonpharmacological approaches 11:20 – Realistic timeline of gene therapies for epilepsy 13:15 – Future plans for research, current unmet needs This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Clears AI-Powered Brain MRI Software Pixyl.Neuro FDA Grants Meeting With BrainStorm Cell Therapeutics on ALS Therapy NurOwn FDA Warns of Serious Adverse Effects for Using Levetiracetam and Clobazam in Epilepsy Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Brent Vaughan, chief executive officer of Cognito Therapeutics, provided insight on the mechanism of action behind the company's gamma sensory stimulation as a potential therapy for patients with Alzheimer disease. He spoke on the positive phase 2 OVERTURE study presented at the 2023 Clinical Trials on Alzheimer's Disease (CTAD) conference, including the notable takeaways from the trial and the safety profile observed. In addition, he commented on how this approach can be used with newer therapies and what the company is looking to achieve in a phase 3 trial. Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page.Episode Breakdown: 1:10 – Overview of phase 2 OVERTURE study 4:45 – Mechanism of action of gamma sensory stimulation 10:00 – Potential therapeutic crossover with emerging treatments 12:30 – Neurology News Minute 14:50 – Feasibility of the stimulation device 18:20– Future plans and planned phase 3 study This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: ATA188 Fails to Meet Primary End Point In Phase 2 EMBOLD Study of Progressive MS Elecsys Neurofilament Light Test Gains Breakthrough Device Designation as Way to Track MS Disease Activity FDA Clears Phase 2 Study of CAR T-Cell Therapy KYV-101 in Myasthenia Gravis Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question.In this episode, we're covering the recent approval of vamorolone (Agamree; Santhera Pharmaceuticals) oral suspension 40 mg/mL for the treatment of patients with Duchenne muscular dystrophy (DMD) aged 2 years and older. The therapy is set to be acquired by Catalyst Pharmaceuticals soon, and is a first-in-class dissociative steroid that aims to retain the anti-inflammatory activity of corticosteroids while decreasing the deleterious adverse events (AEs). We spoke with Barry J. Byrne, MD, PhD, professor and associate chair of pediatrics and director of the Powell Gene Therapy Center at the University of Florida, who discussed the recent FDA approval of Santhera Pharmaceuticals investigational agent vamorolone (now marketed as Agamree, and soon to be acquired by Catalyst Pharmaceuticals in North America), its potential to affect the treatment paradigm as a first-in-class dissociative steroid, and important efficacy and safety information from the phase 2b VISION-DMD study (NCT03439670).For more of NeurologyLive's coverage of vamorolone's approval, head here: FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy Episode Breakdown: 0:30 – Vamorolone approved for Duchenne muscular dystrophy 2:20 – Barry J. Byrne, MD, PhD, on his immediate reaction 3:25 – Safety data on vamorolone 4:00 – Byrne on the importance of bone mineralization in DMD 5:10 – Phase 2b efficacy data from VISION-DMD 5:45 – Byrne with context on the efficacy data 7:05 – Byrne with context on the landscape of DMD therapeutic development and personalization approaches Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.REFERENCES1. Catalyst Pharmaceuticals Reports FDA Approval of AGAMREE® (vamorolone) for Duchenne Muscular Dystrophy Granted to Santhera Pharmaceuticals. News release. Catalyst Pharmaceuticals. October 26, 2023. Accessed October 26, 2023. https://www.globenewswire.com/en/news-release/2023/10/26/2767947/13009/en/Catalyst-Pharmaceuticals-Reports-FDA-Approval-of-AGAMREE-vamorolone-for-Duchenne-Muscular-Dystrophy-Granted-to-Santhera-Pharmaceuticals.html2. Santhera and ReveraGen announce positive and statistically highly significant topline results with vamorolone in pivotal VISION-DMD study. News release. Santhera. June 1, 2021. Accessed February 1, 2023. https://www.globenewswire.com/news-release/2021/06/01/2239124/0/en/Santhera-and-ReveraGen-Announce-Positive-and-Statistically-Highly-Significant-Topline-Results-with-Vamorolone-in-Pivotal-VISION-DMD-Study.html3. Dang U, Guglieri M, Clemens PR, et al. Delayed start analysis of efficacy outcomes in placebo to vamorolone crossover participants in VBP15-004. Presented at MDA Clinical and Scientific Conference; March 13-16. Poster 89.4. Muscular Dystrophy Association-Supported Drug AGAMREE® (vamorolone) Approved for the Treatment of Duchenne Muscular Dystrophy by the FDA. News release. MDA. October 26, 2023. Accessed October 26, 2023. https://www.mda.org/press-releases/mda-supported-drug-agamree-vamorolone-approved-for-the-treatment-of-duchenne-muscular-dystrophy-by-the-fda5. Guglieri M, Clemens PR, Perlman SJ, et al. Efficacy and safety of vamorolone vs placebo and prednisone among boys with Duchenne muscular dystrophy: a randomized clinical trial. JAMA Neurol. Published online August 29, 2022. doi:10.1001/jamaneurol.2022.2480

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Alfonso Fasano, MD, PhD, professor of neurology at the University of Toronto and staff neurologist at Toronto Western Hospital, who spoke on the topic of neuromodulation in Parkinson disease and the potential targets clinicians should be learning about. He discussed the specific patient profiles who fit for neuromodulation, ways to improve this type of personalized medicine, and the needed research in this patient population going forward. Looking for more movement disorder discussion? Check out the NeurologyLive® Movement Disorder clinical focus page. Episode Breakdown: 1:10 – Overview of MDS presentation 2:40 – Reasons for complexities with freezing in parkinsonism 4:35 – Aspects of deep brain stimulation care that need improvement 7:00 – Neurology News Minute 9:15 – Promising neuromodulation targets in the brain 12:35 – Finding optimal candidates for specific neurosurgery procedures 18:50 – Future research of neuromodulation in Parkinson disease This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Eisai Plans to Submit BLA for Subcutaneous Lecanemab After Findings Show Promising Results FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy SRP-9001 Fails to Meet Primary End Point in Phase 3 EMBARK Study Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of zilucoplan (Zilbrysq; UCB Pharma) for the treatment of patients with acetylcholine receptor antibody–positive generalized myasthenia gravis. We spoke with James F. “Chip” Howard, MD, Distinguished Professor of Neuromuscular Disease and professor of neurology and medicine at The University of North Carolina at Chapel Hill School of Medicine, and the lead global investigator of the phase 3 RAISE study (NCT04115293), who discussed the downstream effects of this approval for myasthenia gravis, the available data from clinical studies, and the advantages for patients and physicians. For more of NeurologyLive®'s coverage of zilucoplan's approval, head here: FDA Approves Complement C5 Inhibitor Zilucoplan as Treatment for Myasthenia Gravis Episode Breakdown: 0:30 – Zilucoplan approved for AChR+ myasthenia gravis 1:40 – James F. Howard, MD, on his immediate reaction 3:30 – Safety data from the RAISE trial 4:25 – Howard on the potential advantages of zilucoplan 6:50 – Phase 3 efficacy data for zilucoplan 7:55 – Howard with context on the efficacy and the challenges with therapeutic availability in myasthenia gravis 12:10 – Howard, on the future of therapeutic development Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com. REFERENCES 1. UCB announces US FDA approval of Zilbrysq (zilucoplan) for the treatment of adults with generalized myasthenia gravis. News release. October 17, 2023. Accessed October 17, 2023. https://www.ucb.com/stories-media/Press-Releases/article/UCB-announces-US-FDA-approval-of-ZILBRYSQR-zilucoplan-for-the-treatment-of-adults-with-generalized-myasthenia-gravis 2. Howard JF, Bresch S, Genge A, et al. Safety and efficacy of zilucoplan in patients with generalized myasthia gravis (RAISE): a randomized, double-blind, placebo-controlled, phase 3 study. 2023;22(5):395-406. doi:10.1016/S1474-4422(23)00080-7 3. Genge A, Hussain Y, Kaminski HJ, et al. Safety and tolerability of zilucoplan in RAISE-XT: a multicenter, open-label extension study in patients with myasthenia gravis. Presented at: MDA 2023; February 19-22; Dallas, TX. Abstract 145.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we spoke with a variety of specialists in neurology who presented research and gave talks at MSMilan, the joint ECTRIMS-ACTRIMS meeting, held October 11-13, in Milan, Italy. Those included in this week's episode, in order of appearance, are: Mitzi Joi Williams, MD, founder and chief executive officer of the Joi Life Wellness Group Multiple Sclerosis Center. Rhonda Voskuhl, the director of the Multiple Sclerosis Program at UCLA. Tom Fuchs, MD, PhD, a research fellow at the MS Center at Amsterdam University Medical Center. Barry Singer, MD, the director and founder of The MS Center for Innovations in Care. Georgina Arrambide, a clinical neurologist in the MS Center of Catalunya. Want more from the MSMilan, the joint ECTRIMS-ACTRIMS meeting? Click here for all of NeurologyLive®'s coverage of MSMilan 2023. Episode Breakdown: 1:10 – Joi Williams on expanding the use of biomarkers and the next steps in advancing precision medicine for multiple sclerosis treatment. 2:20 – Voskuhl on the increased research efforts to better understand the links between aging, menopause, and women with MS. 4:30 – Fuchs on the challenges of tracking cognitive decline in multiple sclerosis and improving currently available assessments. 7:50 – Singer on the introduction of biosimilars to MS care, the rigorous approval process they undergo, and the potential benefits they bring to the field. 11:45 – Arrambide on an updated consensus approach to the differential diagnosis of suspected multiple sclerosis, and the red flags clinicians should be aware of during this process. This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Gunter Hoglinger, MD, professor of neurology and translational neuroscientist at Ludwig Maximillian's University Munich, provided commentary on a newly initiated phase 3 study assessing AMX0035 (Relyrvio; Amylyx Pharmaceuticals) in patients with progressive supranuclear palsy (PSP). Otherwise known as the ORION trial, Hoglinger discussed what went into trial inclusion, primary end points, and why they believe this therapy, which was approved for patients with amyotrophic lateral sclerosis (ALS) in 2022, could have benefits in PSP. Looking for more neuromuscular disorder discussion? Check out the NeurologyLive® Neuromuscular clinical focus page.Episode Breakdown: 1:15 – Underlying mechanisms of progressive supranuclear palsy 3:30 – Similarities and differences between ALS and PSP 5:00 – Overview of ORION study design 7:00 – Neurology News Minute 9:15 – Reasons behind specific trial inclusion 10:35 – Primary end points of the study 12:50 – Significance of a potentially successful trial This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Advisory Committee Downvotes Mesenchymal Stem Cell Approach NurOwn as Potential ALS Therapy FDA Approves HoneyNaps’ Artificial Intelligence Sleep Disorder Diagnosis Software FDA Approves Cipaglucosidase Alfa and Miglustat as First Two-Component Therapy for Pompe Disease Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Birgitte R. Kornum, MD, an associate professor in the department of neuroscience at the University of Copenhagen, discussed recently published preclinical research assessing phenotypical features and disease progression of narcolepsy type 1. Kornum provided insight on the 2 mouse models used, the effectiveness of the DVC System, and some of the phenotypic features identified. In addition, she gave insight on how the research adds to the ongoing puzzle of treating narcolepsy and where future efforts should be directed toward.Looking for more movement disorder discussion? Check out the NeurologyLive® Narcolepsy clinical focus page.Episode Breakdown: 1:15 – Overview of Medicare study and objectives 3:15 – Advantages of DVC System 5:40 – Clinical takeaways from the findings 9:30 – Neurology News Minute 12:05 – Critical phenotypic features of narcolepsy 14:20 – Significance of this research, future goals This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Alzheimer Agent ALZ-801 Improves Cognition, Reduces Relevant Biomarker Levels in 2-Year Analysis FDA Updates Label for Psychosis Medication Pimavanserin FDA Approves AI-Incorporated Alzheimer Detection Software AIRAscore Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, James Beck, PhD, senior vice president and chief scientific officer of the Parkinson's Foundation provided perspectives on a recently published Medicare claims study that highlighted major gaps and disparities in access to Parkinson disease care. He spoke on the significance of the findings, including the lack of movement disorder specialists and issues with access to mental health professionals despite high rates of depression in this patient population. Furthermore, he spoke on the ways to make specialized care for Parkinson disease more obtainable, especially for those in rural or underserved communities. Looking for more movement disorder discussion? Check out the NeurologyLive® Movement disorder clinical focus page. Episode Breakdown: 1:15 – Overview of Medicare study and objectives 2:20 – Greatest clinical take-home points from the data 4:15 – Utilizing resources to cover gaps in access 5:30 – Neurology News Minute 8:05 – Reasons for lack of mental health professional support 10:55 – Challenges with understanding depression in PD 12:35 – Ensuring access to PD care in rural/underserved communities This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Sandoz’s Natalizumab-sztn as First Biosimilar for Multiple Sclerosis Duchenne Agent Pamrevlumab Fails to Meet Primary End Point in Phase 3 LELANTOS-2 Trial FDA Issues Ravulizumab Complete Response Letter for NMOSD, Citing Needed Changes to REMS Program Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Nicole Fowler, PhD, director of research for internal medicine and geriatrics at Indiana University School of Medicine, discussed the DAVOS Alzheimer's Collaborative DIGITAL Demonstration Project, an initiative to help understand the feasibility, acceptability, and implementation of digital cognitive screening tools in primary care settings. Specifically, she spoke on how these tools will be used as more novel therapeutics for Alzheimer disease emerge, the challenges with screening patients large-scale, and how blood tests and other biomarkers will play a role going forward. Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page. Episode Breakdown: 1:15 – Objectives of DAVOS Project, overview of presentation from AAIC 6:55 – Complexities and challenges with screening in primary care settings 10:30 – Neurology News Minute 13:25 – Incorporating screening procedures with emerging therapies 12:30 – Future role of digital technologies and blood tests This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Expanded Indication of DaxibotulinumtoxinA to Treat Cervical Dystonia Exon 44 Skipping Agent AOC 1044 Granted FDA Orphan Drug Designation for Duchenne Muscular Dystrophy CGRP Medication Atogepant Gains European Commission Approval as Migraine Preventive FDA Approves Neurocrine Biosciences’ Valbenazine for Huntington Disease Chorea Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question.In this episode, we're covering the recent expanded indication of valbenazine (Ingrezza; Neurocrine Biosciences) to include the treatment of chorea associated with Huntington disease (HD). Erin Furr-Stimming, MD, FAAN, FANA, a professor of neurology at McGovern Medical School of UTHealth Houston, and principal investigator of the phase 3 KINECT-HD studies, valbenazine's supportive studies, offered her immediate reaction to the news. In addition, she spoke about the efficacy observed in these trials, the advantages valbenazine has as a VMAT2 inhibitor, and the remaining unmet needs in the management of HD. For more of NeurologyLive®'s coverage of valbenazine's expanded indication, head here: FDA Approves Neurocrine Biosciences’ Valbenazine for Huntington Disease Chorea Episode Breakdown: 0:30 – Valbenazine approved for Huntington disease chorea 1:35 – Erin Furr-Stimming, MD, FAAN, FANA, on immediate reaction 2:15 – Treatment toolbox for HD chorea 3:05 – Furr-Stimming on mechanistic advantages of valbenazine 5:10 – Phase 3 efficacy data of valbenazine 5:55 – Furr-Stimming on greatest clinical takeaways from trials 6:55 – Current state of Huntington management 8:00 – Furr-Stimming on current unmet needs for patients, including research on disease-modifying therapies Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.REFERENCES1. Neurocrine Biosciences Announces FDA Approval of INGREZZA® (valbenazine) Capsules for the Treatment of Chorea Associated With Huntington's Disease. News Release. Neurocrine Biosciences. Published August 18, 2023. Accessed August 21, 2023.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, Thomas Crawford, MD, a pediatric neurologist at Johns Hopkins Medicine, spoke on the recently published 5-year analysis of the NURTURE study (NCT02386553), a long-term trial assessing the efficacy and safety of nusinersen (Spinraza; Biogen) in presymptomatic infants with spinal muscular atrophy (SMA). Crawford discussed the significance of the positive findings, the shift in conversations around treatment optimization in SMA, and how subgroup data may factor into the design of future trials.Looking for more Neuromuscular Disorders discussion? Check out the NeurologyLive® neuromuscular clinical focus page.Episode Breakdown: 1:20 – Benefits seen with nusinersen in NURTURE 4:10 – Changes in goals for treating SMA 6:05 – Complexities with getting infants therapy days after diagnosis 9:40 – Neurology News Minute 12:30 – Subgroup findings from NURTURE 15:20 – Ways to improve treatment optimization in SMA This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: World Health Organization Adds Several MS Treatments to List of Essential Medicines Ceribell's Status Epilepticus Software Receives FDA Clearance With CMS NTAP Coverage Included FDA Accepts New Drug Application for Long-Acting Form of Glatiramer Acetate Essential Tremor Agent Ulixacaltamide Continues to Show Positive Results in Essential1 Study Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. This episode features an in-depth dive into the recent approval of lecanemab (Leqembi; Eisai) for Alzheimer disease and the ongoing debate about its potential benefit, the amyloid-targeting class of medicines, and the communication of data. Featured in this episode is commentary from 2 experts in neurology clinical care: Anton P. Porsteinsson, MD, the William B. and Sheila Konar Professor of Psychiatry, Neurology, Neuroscience, and Medicine, and the director of the Alzheimer's Disease Care, Research and Education Program at the University of Rochester School of Medicine and Dentistry; and Alberto Espay, MD, PhD, the division director and Research Endowed Chair of the James J. and Joan A. Gardner Family Center for Parkinson’s Disease and Movement Disorders, and a professor of clinical neurology and rehabilitation medicine at the University of Cincinnati. Looking for more Alzheimer disease discussion? Check out the NeurologyLive® dementia and Alzheimer disease clinical focus page. Episode Breakdown: 1:45 – Introduction and main findings 3:15 – Espay, with his perspective on the downstream effects of the approval 10:10 – Porsteinsson, with his perspective on the downstream effects of the approval 18:30 – Secondary end points and safety data 20:05 – Porsteinsson, on the recent progress in AD 23:05 – Espay, on contextualizing the effect of amyloid clearance 26:00 – Porsteinsson, on the logistics of the approval 34:20 – Espay, on the lingering unanswered questions 38:20 – Espay, on the communication of data to the public around the amyloid therapies 42:25 – Porsteinsson, on how things evolve going forward This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories and studies featured in this week's show, as well as additional expert interviews on lecanemab, can be found here: Show notes: Alberto Espay’s lecanemab data infographic Lecanemab CLARITY data Donanameb TRAILBLAZER data Additional reading and coverage: FDA Grants Traditional Approval to Lecanemab as Therapy for Early-Stage Alzheimer Disease Medicare Expands Coverage for Fully Approved Alzheimer Agent Lecanemab Lecanemab to be Tested in Preclinical Alzheimer Disease Therapeutics Aim at Modifying Alzheimer Disease Additional expert interviews: David Bates, PhD: Downstream Impacts of Lecanemab on Alzheimer Disease, Future Drug Development Howard Fillit, MD: Lecanemab’s Impact on Care and Diagnosis and the Future of Alzheimer Disease Treatment Nicole Fowler, PhD, MHSA: Preparing Primary Care Centers for New Wave of Alzheimer Agents Ronald C. Petersen, MD, PhD: Lessons Learned About Clinically Meaningful Benefit from Antiamyloid Trials Sharon Cohen, MD, FRCPC: Significance of Expanding Antiamyloid Therapy Class, Toolbox to Treat Alzheimer Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com. REFERENCES 1. FDA Converts Novel Alzheimer’s Disease Treatment to Traditional Approval. FDA. News release. July 6, 2023. Accessed July 27, 2023. https://www.fda.gov/news-events/press-announcements/fda-converts-novel-alzheimers-disease-treatment-traditional-approval 2. van Dyck CH, Swanson CJ, Aisen P, et al. Lecanemab in early Alzheimer’s disease. N Engl J Med. 2023;388:9-21. doi:10.1056/NEJMoa2212948 3. Sims JR, Zimmer JA, Evans CD, et al. Donanemab in Early Symptomatic Alzheimer Disease: The TRAILBLAZER-ALZ 2 Randomized Clinical Trial. JAMA. Published online July 17, 2023. doi:10.1001/jama.2023.13239

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, we spoke with Jill Farmer, DO, MPH, an assistant professor of neurology and director of the Parkinson’s Disease & Movement Disorder Program at Global Neurosciences Institute, who spoke with us about the current landscape of care for women with Parkinson disease (PD), how the approach to their care can differ from men, the major gaps around education and treatment for these women, and much more.Looking for more Movement Disorder discussion? Check out the NeurologyLive® movement disorder clinical focus page.Episode Breakdown: 1:15 – The presentation of Parkinson disease for women 3:00 – Nonmotor symptoms that can create challenges for diagnosis 4:45 – OFF episodes and dyskinesia in women vs men 7:00 – Being attentive to patient needs in treatment 10:20 – The daily impact of Parkinson disease 13:05 – Neurology News Minute 16:30 – Encouraging women with Parkinson to participate in trials 18:30 – Barriers to care specific to women 20:00 – Communication with women patients 22:20 – Areas of need for research 23:35 – Conversations in areas of limited knowledge 25:20 – Closing thoughts This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Issues Complete Response Letter to Amneal for Parkinson Agent IPX203 FDA Grants Traditional Approval to Lecanemab as Therapy for Early-Stage Alzheimer Disease Medicare Expands Coverage for Fully Approved Alzheimer Agent Lecanemab FDA Approves Image Guided Programming Software for Vercise Deep Brain Stimulation Systems Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we spoke with a variety of specialists in neurology who presented research and gave talks at 4 recent major medical meetings—the Consortium of MS Centers Annual Meeting, the Annual Joint SLEEP Meeting, the Advanced Therapeutics in Movement and Related Disorders Congress, and the American Headache Society’s Annual Scientific Meeting. Those included in this week's episode, in order of appearance, are: Amit Bar-Or, MD, FRCPC, FAAN, FANA, the Melissa and Paul Anderson President’s Distinguished Professor at the Perelman School of Medicine of the University of Pennsylvania. Gavin Giovannoni, MBBCh, PhD, FCP, FRCP, FRCPath, a professor of neurology at Barts and The London School of Medicine and Dentistry. Dayna Johnson, PhD, MPH, MSW, MS, a sleep epidemiologist and assistant professor at the Rollins School of Public Health at Emory University. John Winkelman, MD, PhD, the chief of the Sleep Disorders Clinical Research Program at Massachusetts General Hospital. Laxman Bahroo, DO, a professor of neurology and the residency program director at MedStar Georgetown University Hospital. Ling Wan-Albert, OTD, OTR/L, an assistant professor of occupational therapy at the New York Institute of Technology. Sara Pavitt, MD, the chief of headache at UT Austin Dell Children’s Hospital. Ali Ezzati, MD, the director of the Neuroinformatics Program at the University of California, Irvine. Want more from all of NeurologyLive's coverage of these medical meetings—including dozens more interviews with expert clinicians—click the links below: Consortium of Multiple Sclerosis Centers Annual Meeting SLEEP, the Joint American Academy of Sleep Medicine and the Sleep Research Society Meeting Advanced Therapeutics in Movement and Related Disorders Congress American Headache Society Annual Scientific Meeting Episode Breakdown: 1:15 – Bar-Or on the potential of BTK inhibition in multiple sclerosis at CMSC 2023 4:40 – Giovannoni on the understanding of MS as a smoldering disease at CMSC 2023 10:30 – Johnson on multilevel efforts to address modifiable factors of sleep health at SLEEP 2023 13:05 – Winkelman on moving away from dopamine agonists as first-line treatments for restless legs syndrome at SLEEP 2023 15:30 – Bahroo on the role of botulinumtoxins in Parkinson disease care at ATMRD 2023 19:00 – Wan-Albert on the impact of social isolation on cognition at ATMRD 2023 22:10 – Pavitt on the universal knowledge about pediatric headache for neurologists and nonspecialists at AHS 2023 24:05 – Ezzati on the use of machine learning models to improve treatment optimization in heterogenous migraine at AHS 2023 This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of SRP-9001, or delandistrogene moxeparvovec (Elevidys; Sarepta) in Duchenne muscular dystrophy (DMD). Offering commentary is Natalie Goedeker, CPNP, a nurse practitioner in neurology in the Neuromuscular Division at Washington University in St Louis. SRP-9001 is an AAV vector-based gene therapy that was approved for the treatment of ambulatory patients with DMD with a confirmed mutation in the DMD gene aged 4 to 5 years, based on data from the phase 3 EMBARK study (NCT05096221). For more of NeurologyLive®'s coverage of SRP-9001's approval, head here: FDA Approves SRP-9001 as First Gene Therapy for Duchenne Muscular Dystrophy Episode Breakdown: 0:30 – SRP-9001 (Elevidys; Sarepta) is approved for DMD 1:35 – Natalie Goedeker, CPNP, on the approval 3:05 – Efficacy of SRP-9001 4:00 – Goedeker on pieces of efficacy data released 4:45 – Currently available therapies for DMD 5:35 – Goedeker on role of SRP-9001 in treatment landscape 6:55 – Safety data of SRP-9001 8:00 – Goedeker on safety considerations of which prescribing clinicians should be aware Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com. REFERENCES 1. Sarepta Therapeutics announces FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy. News release. June 22, 2023. Accessed June 22, 2023. https://www.businesswire.com/news/home/20230622454844/en/

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, we spoke with Ann Marie Morse, DO, FAAN, a pediatric neurologist and sleep medicine specialist at Geisinger Medical Center, who sat down with NeurologyLive at the annual SLEEP meeting to discuss the landscape of challenges narcolepsy-related challenges that patients face, the influence of gut health on sleep apnea, how nighttime disruptions can affect treatment decisions, emerging concepts of care, and much more.Looking for more Sleep Disorder discussion? Check out the NeurologyLive® sleep disorder clinical focus page.Episode Breakdown: 1:15 – Factors at play in obstructive sleep apnea 3:40 – Literature on the gut microbiome's role in sleep 5:55 – Lingering needs in narcolepsy treatment 8:30 – Neurology News Minute 12:40 – The effect of disrupted nighttime sleep 15:45 – Emerging concepts in sleep medicine This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Biogen and Denali Terminate Phase 3 LIGHTHOUSE Study of BIIB122 in LRRK2-related Parkinson Disease FibroGen's Duchenne Muscular Dystrophy Antibody Pamrevlumab Falls Short in Phase 3 Phase 2/3 Study of Blarcamesine in Pediatrics With Rett Syndrome Completes Enrollment FDA Places Hold on Phase 1 Study of Exon Skipping Therapy PGN-EDODM1 in Myotonic Muscular Dystrophy FDA Panel Votes in Favor of Eisai’s Lecanemab for Early Alzheimer Disease Post-Marketing Study of Andexanet Alfa Stopped Early After Achieving Prespecified Efficacy Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we spoke with Brandon Giglio, MD, a vascular neurologist and clinical assistant professor of neurology at NYU Grossman School of Medicine, as well as the director of Vascular Neurology at NYU Langone Hospital in Brooklyn, New York. As May is annually designated as Stroke Awareness Month in the United States, Giglio sat down with us to extend that time of awareness and share his insight into the current public understanding of stroke, how effective the care paradigm is at prevention and poststroke care, and much more. Looking for more Stroke discussion? Check out the NeurologyLive® stroke clinical focus page. Episode Breakdown: 1:15 – Breaking down the signs and symptoms of stroke with B.E. F.A.S.T 2:45 – The importance of stroke awareness 3:55 – Treating patients with poststroke symptoms 5:45 – Neurology News Minute 10:40 – Notable risk factors for stroke 13:15 – The critical need for novel education tools in stroke This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Potential Path for Phase 3 Study of AOC 1001 in Myotonic Dystrophy Becomes Clear After FDA Ease’s Clinical Hold FDA Grants Fast Track Designation for Atrial Fibrillation Therapy Asundexian FDA Pushes Back Decision Date for Duchenne Gene Therapy SRP-9001 FDA Accepts NDA for DHE Nasal Powder Product STS101 for Acute Migraine CMS Announces Plan to Provide Coverage of Antiamyloid Therapies for Alzheimer Disease Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we spoke with James Berry, MD, PhD, director of the Massachusetts General Hospital ALS Care Center. At the 2023 American Academy of Neurology Annual Meeting, he spoke about new data from a study assessing CNM-Au8, a nanocrystal formulation in development for patients with ALS and other neurological diseases. Additionally, he talked about the current unmet needs in the ALS community, including the need for more diverse treatment options and increased research. Looking for more Multiple Sclerosis discussion? Check out the NeurologyLive® neuromuscular clinical focus page. Episode Breakdown: 1:15 – Clinical development program of CNM-Au8 3:40 – Safety findings, treatment responses of CNM-Au8 4:20 – Next steps in research 6:25 – Neurology News Minute 8:45 – Conducting safe trials 10:00 – Remaining unmet needs for patients with ALS 11:15 – Need for additional research This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Brexpiprazole as First Therapy for Alzheimer Agitation FDA Panel Votes in Favor of Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy in Tight Decision Alzheimer Agent COYA 301 Halts Cognitive Decline, Restores Regulatory T Cell Dysfunction in Phase 1 Study Phase 2 Data Highlight Fenebrutinib's Impact on Brain Lesions in Relapsing Multiple Sclerosis Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com. REFERENCES 1. Berry J, Maragakis N, Paganoni S, et al. Evidence for Survival Benefit in ALS with CNM-Au8 Treatment Across Three Study Populations. Presented at: 2023 AAN Annual Meeting; April 22-27, Boston, Massachusetts.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, we spoke with a variety of specialists in neurology who presented research and gave talks at the 75th American Academy of Neurology Annual Meeting, held April 22-27, 2023, in Boston, Massachusetts. Those included in this week's episode, in order of appearance, are: Michael H. Barnett, MBBS, PhD, FRACP, a consultant neurologist at Royal Prince Alfred Hospital (RPAH) Sydney, director of the RPAH MS Clinic and the MS Clinical Trials Unit at the Brain and Mind Centre; codirector of the MS Research Australia Brain Bank; and a senior professor at the University of Sydney. Nancy R. Foldvary-Schaefer, DO, FAAN, the director of the Sleep Disorders Center and staff in the Epilepsy Center at Cleveland Clinic, and a professor of medicine at the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University. Sean J. Pittock, MD, the director of the Center for Multiple Sclerosis and Autoimmune Neurology and director of the Neuroimmunology Research Laboratory at Mayo Clinic. Katherine W. Turk, MD, a neurologist at Boston VA Memory Disorders Clinic, principal investigator and codirector of the Center for Translational Cognitive Neuroscience lab (CTCN) at VA Boston; and assistant professor of neurology and coleader of the Outreach, Recruitment and Engagement core of the Alzheimer’s Disease Research Center at Boston University. Erika U. Augustine, MD, MS, the associate chief science officer and director of the Clinical Trials Unit at the Kennedy Krieger Institute. Jeffrey M. Statland, MD, a neuromuscular disease specialist and professor of neurology at the University of Kansas Medical Center. Chian-Chun Chiang, MD, a stroke and migraine specialist and assistant professor of neurology at Mayo Clinic. Want more from the 75th American Academy of Neurology Annual Meeting? Click here for all of NeurologyLive®'s coverage of AAN 2023.Episode Breakdown: 1:10 – Barnett on the topline findings for CNM-Au8 from the VISIONARY-MS clinical trial. 8:35 – Foldvary-Schaefer on the understanding of the relationship between epilepsy and sleep. 13:25 – Pittock on the latest data on ravulizumab from the CHAMPION-NMOSD trial. 19:00 – Turk on the landscape of diagnosis and treatment for mild cognitive impairment, and how to improve the process. 22:00 – Augustine on Dr. Sidney Carter and the current paradigm of care in child neurology. 27:15 – Statland on the findings for ataluren in nonsense mutation Duchenne muscular dystrophy from Study 041. 34:05 – Chiang on the takeaways from a big data analysis of 25 therapies for acute migraine management. This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.REFERENCES1. Barnett MH. VISIONARY-MS Top-line Results: A Phase 2, Randomized, Double-Blind, Parallel Group, Placebo-controlled Study to Assess the Safety and Efficacy of CNM-Au8, a Catalytically Active Gold Nanocrystal Suspension in Relapsing Multiple Sclerosis. Presented at: AAN Annual Meeting; April 22-27, 2023; Boston, MA.2. Foldvary-Schaefer NR. Diagnostic Testing: Beyond the MSLT. Presented at: AAN Annual Meeting; April 22-27, 2023; Boston, MA.3. Pittock SJ. Efficacy and safety of ravulizumab in adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder: outcomes from the phase 3 CHAMPION-NMOSD trial. Presented at: AAN Annual Meeting; April 22-27, 2023; Boston, MA.4. Statland JM. Safety and Efficacy of Ataluren in nmDMD Patients from Study 041, a Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial. Presented at: AAN Annual Meeting; April 22-27, 2023; Boston, MA.5. Chiang CC. Simultaneous Comparisons of 25 Acute Migraine Medications: A Big Data Analysis of 10 Million Patient Self-Reported Treatment Records From A Migraine Smartphone Application. Presented at: AAN Annual Meeting; April 22-27, 2023; Boston, MA.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question.In this episode, we're covering the recent approval of tofersen (Qalsody; Biogen/Ionis) in SOD1 mutation-mediated amyotrophic lateral sclerosis (ALS). Offering commentary is Angela Genge, MD, FRCPC, the director of the ALS Centre of Excellence for Research and Patient Care at McGill University, and an executive at the Clinical Research Unit at The Neuro (Montreal Neurological Institute-Hospital). Tofersen is an antisense oligonucleotide, and its new drug application (NDA) was supported by data from a phase 1 study of healthy volunteers, a phase 1/2 dose-ascending study, the pivotal phase 3 VALOR study (NCT02623699), and its open-label extension.For more of NeurologyLive®'s coverage of tofersen's approval, head here: FDA Approves Tofersen as First SOD1-ALS Treatment Episode Breakdown: 0:30 – Tofersen (Qalsody; Biogen/Ionis) approved for SOD1-ALS 1:35 – Angela Genge, MD, FRCPC, on the approval 3:05 – The implications of targeting a root cause of ALS 5:00 – Data from the supporting VALOR study 5:55 – Genge on the context around the trial data 7:35 – Questions about access to tofersen amid accelerated approval Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.REFERENCES1. FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene. News release. FDA. April 25, 2023. Accessed April 25, 2023. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene2. Miller TM, Cudkowicz ME, Genge A, et al. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2022;387:1099-1110. doi:10.1056/NEJMoa2204705.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we spoke with Robert Zivadinov, MD, PhD, director of the Buffalo Neuroimaging Analysis Center, and a professor of neurology and biomedical informatics at the Jacobs School of Medicine & Biomedical Sciences at the University at Buffalo. He discussed the recent CASA-MS study that he and colleagues conducted into a subgroup of patients with multiple sclerosis (MS) who develop rapid and progressive disability at a relatively young age, which he presented earlier this year at the 2023 Americas Committee for Treatment and Research in MS Forum, and the critical effectively need to treat this group of patients. The study was carried out at The Boston Home, a specialized residential facility for individuals with advanced progressive neurological disorders, in Dorchester, Massachusetts, and at the University at Buffalo, in New York. Looking for more Multiple Sclerosis discussion? Check out the NeurologyLive® multiple sclerosis clinical focus page. Episode Breakdown: 1:10 – Background on the CASA-MS study subpopulation 3:45 – Topline results of the study 6:05 – The need for further clarification in MS populations 7:10 – The utility of biomarkers such as GFAP 12:15 – Further assessment plans for the CASA-MS cohort 14:15 – Neurology News Minute 18:35 – Access to the tools to characterize progressive MS 21:30 – The potential with portable MRI and artificial intelligence 25:25 – Unmet needs in progressive MS This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Phase 3 Study of GTX-104 in Subarachnoid Hemorrhage Expected to Proceed Following FDA Feedback FDA Places Partial Hold on Evobrutinib Initiation in Newly Enrolled Patients With MS FDA Panel Votes in Favor of Brexpiprazole as Potential Therapy for Alzheimer Agitation FDA Approves IntelGenx and Gensco's Rizatriptan for Acute Migraine Treatment DEA Rules Fenfluramine No Longer a Controlled Substance Atogepant’s Indication Expanded to Include Prevention of Chronic Migraine FDA Grants Breakthrough Device Designation to Reach Neuro’s Chronic Stroke Platform FDA Approves CSL Behring's Immune Globulin Subcutaneous Prefilled Syringe for PI and CIDP Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com. REFERENCE 1. Zivadinov R, Jakimovski D, Burnham A, et al. Comprehensive Assessment of Severely Affected Multiple Sclerosis (CASA-MS) Study. Presented at: ACTRIMS Forum; February 25-28, 2023; San Diego, CA. P430. www.abstractsonline.com/pp8/#!/10822/presentation/468

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we spoke with Dilesh Doshi, PharmD, vice president of Health Economic and Outcomes Research at Acadia Pharmaceuticals. He spoke on the advantages and benefits of pimavanserin (Nuplazid; Acadia), the only FDA-approved therapy to treat Parkinson disease psychosis. Additionally, he discussed recent data that compared efficacy and safety of other off-label therapies such as clozapine and quetiapine. Looking for more Parkinson disease discussion? Check out the NeurologyLive® movement disorder clinical focus page. Episode Breakdown: 1:10 – Mechanistic differences between pimavanserin and other atypical antipsychotics 3:00 – Understanding drug-to-drug interactions for PDP 5:05 – Decision making with quetiapine and negative cognitive effects 7:10 – Improved use of pimavanserin since approval 9:00 – Neurology News Minute 11:40 – Potential predictors of mortality in Parkinson disease psychosis 15:20 – Possibility of preventing PD psychosis 17:20 – Future research on identifying subgroups of responders to treatment This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Tiziana Announces Planned Phase 2 Study of Foralumab in Progressive Multiple Sclerosis Blarcamesine Deemed Safe, Effective in Phase 2 Extension Study of Parkinson Disease Dementia Eplontersen Continues to Show Clinical Benefit in Long-Term Analysis of hATTR Polyneuropathy Reldesemtiv to be Discontinued in ALS Following Data Monitoring Committee Analysis Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, we spoke with Eric Zhou, PhD, assistant professor of pediatrics at Harvard Medical School. He spoke on a recently conducted study assessing the link between narcolepsy in adolescents and social relationship health. Specifically, he talked about how the sleep disorder impacts mood, physical activities, driving, and other long-term health outcomes, as well as ways we can help adolescents with narcolepsy at the earliest stages. Looking for more narcolepsy discussion? Check out the NeurologyLive® narcolepsy clinical focus page.Episode Breakdown: 1:20 – Identified links between narcolepsy and social relationships 3:05 – Long-term impacts of affected mood, physical activities, and driving 4:15 – Current assessment of narcolepsy in adolescents 5:30 – Neurology News Minute 8:05 – Attempts to prevent narcolepsy at earliest stages 9:15 – Implementing programs in school to help adolescents with narcolepsy 11:20 – Future research on narcolepsy in younger populations This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA to Hold AdComm Meeting for Investigational DMD Treatment SRP-9001 Treg Suppressing Agent COYA 302 Shows Slowing of ALS Disease Progression FDA Issues Complete Response Letter for AbbVie’s 24-Hour Continuous Carbidopa/Levodopa Pump FDA AdComm Panel Supports Neurofilament Light as Surrogate Biomarker for Tofersen, With Questions Regarding Clinical Benefit Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question.In this episode, we're covering the recent approval of trofinetide (Daybue; Acadia) in Rett syndrome. Offering commentary is Jeffrey L. Neul, MD, PhD, the Annette Schaffer Eskind Chair and director of the Vanderbilt Kennedy Center, and professor of pediatrics at Vanderbilt University Medical Center, and the lead investigator on the phase 3 study of the drug, called LAVENDER (NCT04181723). Trofinetide is a synthetic analog of the amino‐terminal tripeptide of IGF-1, and is now the first treatment approval for Rett syndrome, marking a milestone in the field of developmental disorder care.For more of NeurologyLive®'s coverage of trofinetide's approval, head here: FDA Approves Acadia's Trofinetide for Rett Syndrome Episode Breakdown: 0:30 – Trofinetide (Daybue; Acadia) approved for Rett syndrome 1:30 – Jeffrey L. Neul, MD, PhD, on the approval 4:10 – Prevalence of Rett syndrome and the impact on research 5:40 – Supporting data from the phase 3 LAVENDER study 7:20 – Safety data from the clinical development and managment of common adverse events 8:45 – Closing thoughts Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.REFERENCES1. Acadia Pharmaceuticals Announces U.S. FDA Approval of DAYBUE™ (trofinetide) for the Treatment of Rett Syndrome in Adult and Pediatric Patients Two Years of Age and Older News Release. Acadia Pharmaceuticals. March 10, 2023. Accessed March 13, 2023. https://acadia.com/media/news-releases/acadia-pharmaceuticals-announces-u-s-fda-approval-of-daybue-trofinetide-for-the-treatment-of-rett-syndrome-in-adult-and-pediatric-patients-two-years-of-age-and-older/2. Neul JL. Rett Syndrome. National Organization for Rare Diseases. Updated December 17, 2019. Accessed March 13, 2023. https://rarediseases.org/rare-diseases/rett-syndrome/

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this episode, we spoke with Elizabeth “Eli” Pollard, vice president & chief education and training officer at the Parkinson's Foundation, and the executive director of the World Parkinson Coalition. She spoke about the foundation's ongoing efforts in providing education about Parkison disease (PD), the challenges of managing a growing population of patients, the need for community physicians and specialists to work in unison, and more.>>Click here to access the Parkinson Foundation's educational series.Looking for more Parkinson disease discussion? Check out the NeurologyLive® PD disease spotlight page.Episode Breakdown: 1:20 – The need for education across the PD care paradigm 4:40 – An overview of the 6-part educational series 6:10 – The inclusion of specialties such as dentistry in the care of PD 9:40 – Using training to overcome gaps in multidisciplinary care 13:05 – Challenges in educating a large population of providers 16:55 – Neurology News Minute 19:00 – Improving access to clinical education 24:07 – The Foundation’s goal to inspire learning to improve care 26:30 – Closing thoughts This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com.The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Theranica's Nerivio for Preventive Treatment of Migraine FDA Approves Omaveloxolone As First Treatment for Friedreich Ataxia FDA Accepts Supplemental BLA for Traditional Approval of Lecanemab FDA Clears Abbott’s Alinity i Lab-Based Blood Test for Traumatic Brain Injury Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we spoke with Darin Okuda, MD, director of Neuroinnovation and Multiple Sclerosis & Neuroimmunology Imaging Program at UT Southwestern Medical Center. He spoke about the phase 2 ARISE study assessing dimethyl fumarate (Tecfidera; Biogen) in patients with radiologically isolated syndrome, the significance of the findings, next steps in validating this therapy, and more. Looking for more multiple sclerosis discussion? Check out the NeurologyLive® multiple sclerosis clinical focus page. Episode Breakdown: 1:20 – Overview of ARISE and conduct of study 4:20 – Potential subgroup responders to dimethyl fumarate 8:10 – Efforts to improve the detection of radiologically isolated syndrome 10:05 – Neurology News Minute 11:55 – Next steps of research following ARISE 16:50 – Closing thoughts on significance of the study This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: CNM-Au8 Shows Positive Findings on MS-Related Biomarkers of Whole Brain, White Matter Aducanumab’s Label Updated to Include Risks on Brain Bleeding Primary Care Physicians Show Greater Confidence in Diagnosing Alzheimer Disease Through DISCERN Skin Test FDA Approves Extended-Release Deutetrabenazine for Huntington Disease Chorea, Tardive Dyskinesia Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com. REFERENCE Okuda DT, Kantarci O, Lebrun-Frenay C, et al. Dimethyl fumarate delays multiple sclerosis radiologically isolated syndrome. Ann Neurol. 2023;93(3):604-614. doi:10.1002/ana.26555