Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Donna Shipp, a Boston-area nurse who was misdiagnosed with cancer, underwent surgery and later realized she had IgG4-RD. She now advocates on behalf of others with this rare disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Abby Bronson, vice president of patient advocacy at Edgewise Therapeutics, about a new effort to raise awareness of Becker muscular dystrophy as a disease distinct from Duchenne muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Allison Moore, founder and CEO of the Hereditary Neuropathy Foundation. Moore was the winner of the Muscular Dystrophy Association's 2026 Donavon Decker Legacy Award for Community Impact in Research.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Crowley, president and CEO of the Biotechnology Innovation Organization (BIO), founder of Amicus Therapeutics, and father of 2 children with Pompe disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Hyun Kim, MD, professor of medicine and director of the University of Minnesota's Interstitial Lung Disease Program, about idiopathic pulmonary fibrosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Andrea Wilson Woods, founder of Blue Faery: The Adrienne Wilson Liver Cancer Association—a nonprofit that advocates for patients with hepatocellular carcinoma.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michael Shilsky, MD, of the Yale School of Medicine, on potential therapies for Wilson disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Yen Chen, PhD, of the University of Michigan, on the subject of cognitive dysfunction or "brain fog" among people with scleroderma.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Danish hepatologist Aleksander Krag, MD, PhD, on the diagnosis and treatment of alpha-1 antitrypsin deficiency (AATD).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Vesna Aleksovska, chair of the nonprofit organization Life With Challenges. Aleksovska, who has Gaucher disease, advocates for all rare disease patients in North Macedonia, a former Yugoslav republic.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Wes Michael, founder of Rare Patient Voice. Since 2013, RPV has offered patients and caregivers opportunities to share their input with companies developing products to improve lives.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Katharine Provencher, director of patient advocacy at IgG4Ward!, a nonprofit that advocates for patients and caregivers affected by IgG4-RD.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Cecilia Dueñas, PsyD, on the PBC Research Foundation, and the discrimination and stigma she faced for years before getting diagnosed with the disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Golie-Lorenzo Green, whose lifelong battle with sickle cell disease led him to advocate for others with the debilitating illness.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jana Monaco, a NORD patient ambassador from Virginia who's made nationwide newborn screening her life's mission.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Alabama computer scientist Matthew Might, PhD, on how his son's death from a rare genetic disease led him to become an expert in AI and precision medicine.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sarah Chang, PhD, about the patient and caregiver burden associated with the ultra-rare mitochondrial disease TK2d.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews rheumatologist Jinoos Yazdany, MD, on the potentially catastrophic medical, ethical and legal consequences of relying too much on AI.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Barry Byrne, MD, PhD, an expert on Pompe disease and director of the University of Florida's Powell Gene Therapy Center.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Eric Matteson, MD, of the Mayo Clinic and winner of the American College of Rheumatology's 2025 Presidential Gold Medal.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Karen Smoot, a resident of New Mexico who has both idiopathic pulmonary fibrosis and pulmonary arterial hypertension. Karen is a patient ambassador for the Pulmonary Fibrosis Foundation.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Gabriela Romanow, founder of Boston, Massachusetts-based Rare Vision. The project showcases works by artists with rare neuroimmune diseases such as NMOSD, MOGAD and transverse myelitis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lisa Shea, director of global patient advocacy and engagement for immunology at Johnson & Johnson, about the experiences of patients affected by HDFN and FNAIT.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Nicole Lamanna, MD, a leukemia physician specializing in CLL treatment at Columbia University Medical Center in New York City.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews May Lee Tjoa, PhD, senior global medical affairs leader for Johnson & Johnson's nipocalimab and maternal-fetal immunology division.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Mascarenhas, MD, a professor with the Icahn School of Medicine at Mount Sinai in New York, on advancing care in myelofibrosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Catherine Miller, PharmD, global medical affairs lead for hereditary angioedema (HAE) at Intellia Therapeutics, on an experimental gene editing therapy known as lonvo-Z.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Shoshana Revel-Vilk, MD, director of the Gaucher unit at Shaare Zedek Medical Center in Jerusalem, Israel.

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.