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.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jenny Huang, whose son, Benjamin Lou, is an award-winning math major at MIT who also has spinal muscular atrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Omer Abdul Hamid, MD, a neurologist at Nemours Children's Hospital in Orlando, Florida, on gene therapy's potential to treat Duchenne muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Pamela Gavin, the newly appointed CEO of the National Organization for Rare Disorders (NORD).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Patricia Weltin,founder and CEO of Beyond the Diagnosis—a nonprofit that uses art to raise public awareness of children with ultra-rare diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sarah Glass, PhD, chief operating officer of the n-Lorem Foundation—and the mother of a boy with an ultra-rare disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dimitrios Karussis, MD, PhD, who heads the Multiple Sclerosis Center at Israel's Hadassah University Hospital in Jerusalem.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Mary Morlino, who has sarcoidosis and is the patient navigator for the Undiagnosed Diseases Network Foundation.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Tracy Sharp at the Muscular Dystrophy Association's 2024 Clinical & Scientific Conference in Orlando, Florida. Sharp, 70, has Lambert-Eaton myasthenic syndrome (LEMS), a very rare and debilitating neuromuscular disease.