Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Karin Hoelzer, DVM, PhD, senior director of policy and regulatory affairs at the National Organization for Rare Disorders (NORD). Dr. Hoelzer discusses the importance of convincing the US Congress to renew the Rare Pediatric Disease Priority Review Voucher Program, which is set to expire on September 30, 2024.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Susan Ward, PhD, founder and executive director of the Collaborative Trajectory Analysis Project (cTAP), which seeks to broaden patient eligibility for clinical trials in Duchenne muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Taylor Kane, founder and executive director of 'Remember the Girls.' The charity advocates for female carriers of X-linked rare disorders including Duchenne muscular dystrophy and hemophilia.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews neurologist Jeffrey A. Cohen, MD, of Ohio's Cleveland Clinic on the latest developments in cellular therapies to treat multiple sclerosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews patient advocate Maureen Juip of Grosse Pointe, Michigan. Juip, newly appointed secretary of the Friedreich's Ataxia Research Alliance (FARA), is also the mother of 2 children with the rare neuromuscular disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jacquelyn Bainbridge, DPharm, a neurology professor at the University of Colorado in Aurora, on the benefits of medical cannabis in treating multiple sclerosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michelle C. Werner, CEO of Alltrna, on nonsense mutations in Duchenne muscular dystrophy. She's also the mother of a boy with the disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Flavia Nelson, MD, director of the Multiple Sclerosis Center of Excellence at the University of Miami in Florida, on the difficulties of diagnosing neuromyelitis optica spectrum disorder.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kevin M. Flanigan, MD, director of the Center for Gene Therapy at the Abigail Wexner Research Institute of Nationwide Children's Hospital in Columbus, Ohio. The subject of our talk is exon skipping therapies for Duchenne muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Durresamin Khan, the Pakistani mother of a boy with Duchenne muscular dystrophy, about the difficulties of obtaining treatment for DMD patients who are ineligible for gene therapy.