Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Nikki McIntoch, mother of a boy with spinal muscular atrophy (SMA) and founder of the advocacy group Rare Mamas.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Brown, MD, PhD, director of the CLL Center at Dana-Farber Cancer Institute in Boston, Massachusetts, and winner of the 2025 Michael J. Keating Outstanding Achievement Award.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews neurologist Michio Hirano, MD, of Columbia University in New York about an investigative therapy that shows promise in treating thymidine kinase 2 deficiency (TK2d), an ultrarare disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews cardiologist Pradeep P.A. Mammen, MD, of the University of Kansas School of Medicine, on the growing awareness of girls affected by Duchenne muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kfir Oved about the latest Israeli research on myasthenia gravis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ileen Colin del Río, the mother of a boy with Duchenne muscular dystrophy and president of the Cancún-based nonprofit group Duchenne Mexico.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Yuva Gambhir, 22, who doesn't let Duchenne muscular dystrophy get in the way of his aspiring career as a DJ.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Richard Nowak, MD, director of the Myasthenia Gravis Clinic at Yale School of Medicine in New Haven, Connecticut.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Robert Steen of Oslo, Norway. Steen and his wife, Trude, are the parents of Mats Steen, who died of Duchenne muscular dystrophy (DMD) in 2014. An award-winning documentary, The Remarkable Life of Ibelin, is based on their son's story.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Carolina Barnett-Tapia, MD PhD, an associate professor of neurology at the University of Toronto, about how myasthenia gravis is treated throughout the Western Hemisphere.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews British scientist Angela Vincent, winner of the first-ever Lifetime Achievement Award from the Myasthenia Gravis Foundation of America.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Daniel Grant, MD, vice president and global program head of neuroscience and gene therapy at Swiss pharma giant Novartis, on emerging therapies for SMA.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ashley Stanley-Copeland, MD, of Dell Children's Medical Center in Austin, Texas, on therapeutic options available to patients with Duchenne muscular dystrophy (DMD).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ashley Stanley-Copeland, MD, of Dell Children's Medical Center in Austin, Texas, on therapeutic options available to patients with Duchenne muscular dystrophy (DMD).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric neurologist Sithara Ramdas, MD, of Oxford Children's Hospital in England, on neonatal and juvenile myasthenia gravis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lyza Weisman, who has spinal muscular atrophy type 2, about how she copes with progressive loss of muscle function.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Marcus Delatte, PhD, on how the versatile cannabis plant offers innovative treatments for children with Dravet syndrome and Lennox-Gestaut syndrome.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lucy Culp, vice-president of state government affairs at the Leukemia & Lymphoma Society, on top issues of importance to patients with rare blood cancers.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews amyloidosis patient and former basketball player Dan Lier, a partner with the nonprofit group Somebody To Talk To, about his new "Rare Disease Playbook."

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lauren Holder, an activist and patient with Huntington disease, as well as producer and host of the podcast series Help 4 HD Live.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Antonella Favit-Van Pelt, MD, PhD, chief medical officer at Helius Medical, on the potential of neuromodulation to treat people with multiple sclerosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Joyce Kullman, executive director of the Vasculitis Foundation, duirng Vasculitis Awareness Month.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Chris Lewis, whose father, Jerry Lewis, was not only among the most famous comedians of the 20th century but also a tireless advocate for people with muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lisa Butler, CEO of the GBS-CIDP Foundation, on what her organization is doing for patients with chronic inflammatory demyelinating polyneuropathy (CIDP).

Larry Luxner, senior correspondent for Rare Disease Advisor, talks to Donavon Decker, who has limb-girdle muscular dystrophy. Decker is the winner of the 2025 MDA Legacy Award for Community Impact in Research.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews 17-year-old Lily Sander with Charcot-Marie-Tooth disease. The Muscular Dystrophy Association has named Sander its 2025 MDA Patient Ambassador.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Katherine Mathews, MD, winner of the Muscular Dystrophy Association's 2025 Legacy Award for Achievement in Clinical Research for her contributions to understanding and treating genetic disorders affecting the neuromuscular system.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Arash Mostaghimi, MD, an associate professor of dermatology and a practicing physician at Boston's Brigham and Women's Hospital. Dr. Mostaghimi is a recognized expert on generalized pustular psoriasis (GPP).

Rare Disease Advisor correspondent Tori Rodriguez interviews clinical hematologist and professor Luke Chen, MD, about IgG4-related disease and how it mimics various malignancies.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Anderson, MD, chief research officer at AllerVie Clinical Research, on the complexities of diagnosing and treating hereditary angioedema.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Art Still, former defensive end for the Kansas City Chiefs and an NFL Hall of Famer who's now a patient advocate dedicated to raising awareness about amyloidosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews oncologist-hematologist Heather Landau, MD, of Sloan Kettering Memorial Cancer Center in New York City, for National Amyloidosis Awareness Month.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Timothy Craig, DO, a tenured professor of medicine, pediatrics and biomedical sciences at Pennsylvania State University in Hershey. Dr. Craig has been researching hereditary angioedema for 30 years.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sangeetha Venugopal, MD, assistant professor of clinical medicine at the University of Miami's Sylvester Comprehensive Cancer Center. Dr. Venugopal speaks on how smoking worsens outcomes for patients with a group of blood cancers known as myelodysplastic syndromes (MDS).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Andrea Renzi, a New Hampshire mother and patient advocate whose family has been affected by hemolytic disease of the fetus and newborn (HDFN).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews James Bussel, MD, emeritus professor of pediatrics, medicine, and obstetrics and gynecology at New York's Weill Cornell Medicine. The topic of their discussion is nipocalimab as a potential treatment for fetal and neonatal alloimmune thrombocytopenia (FNAIT).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Rigoberto Garcia, executive director of the Hemophilia Foundation of Southern California, about the difficulty minorities—especially undocumented Hispanics—have in accessing treatment for their bleeding disorders.

Larry Luxner, senior correspondent for Rare DIsease Advisor, interviews Kristin Hatcher, director of pediatric and rare diseases at the Global Liver Institute. Hatcher has both alpha-1 antitrypsin deficiency (AATD) and Von Willebrand disease, a rare clotting disorder.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Terry Jo Bichell, PhD, founder and director of CombinedBrain, which advocates for patients with 110 neurogenetic diseases. Dr. Bichell is part of a panel that's studying how the US Food and Drug Administration can do a better job of speeding up clinical trials and becoming more transparent about its decisions.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Erin Collins, founder of the Chasing Rainbows Foundation in North Carolina's Outer Banks. The charity raises awareness of HDFN and other rare pregnancy-related diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dean Wingerchuk, MD, speaks on the potential for treating neuromyelitis optica spectrum disorder (NMOSD) with stem-cell therapy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jimmy Pollard, who for the past 38 years has advocated on behalf of people around the world affected by Huntington disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews clinical psychologist Rosalind Kalb, lead senior programs consultant for Can-Do MS, a Colorado-based nonprofit health and wellness organization.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Tanita Allen, a Black woman with Huntington disease who has made it her mission to increase awareness about the disorder and its devastating impact on patients regardless of their racial or ethnic background.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jean Elwing, MD, a professor of medicine and director of the Pulmonary Hypertension Program at Ohio's University of Cincinnati.

Larry Luxner, senior correspondent for Rare DIsease Advisor, interviews Payel Gupta, MD, on social media platforms such as TikTok, Facebook and YouTube, and their importance in reaching today's public—and fighting online misinformation.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sandy Siegel, founder and president of the Siegel Rare Neuroimmune Association. Siegel started the charity 30 years ago with his wife, Pauline, after she was diagnosed with transverse myelitis. After Pauline's death in 2017, the organization expanded to include five other rare neuroimmune diseases including neuromyelitis optica spectrum disorder (NMOSD).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Anastasia Vishnevetsky, MD, MPH, of Massachusetts General Hospital and Harvard Medical School. Dr. Vishnevetsky is heading a groundbreaking clinical trial of the cannabinoid spray nabiximols in treating the symptoms of NMOSD.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ignacio Muñoz-Sanjuan, PhD, the president and founder of Factor-H—a Los Angeles-based nonprofit organization that works to alleviate extreme poverty among people with Huntington disease in Venezuela, Colombia, and Peru.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Amy Case, MD, chief medical officer of the Pulmonary Fibrosis Foundation, about the importance of passing the bipartisan Supplemental Oxygen Access Reform (SOAR) Act, which is currently stalled in Congress.