Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Steve Van Wormer, cofounder and director of the Phaware Global Association. Van Wormer became a patient advocate after his late son, Lucas, was diagnosed with pulmonary hypertension at age 4.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Victor Test, MD, an expert in pulmonary arterial hypertension (PAH). Dr. Test is a professor of medicine and chair of the pulmonary vascular disease program at Texas Tech School of Medicine in Lubbock.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pulmonologist Drew Harris, MD, an associate professor of medicine at the University of Virginia in Charlottesville. Dr. Harris is also medical director of the Black Lung Program at Stone Mountain Health Services, in the heart of southwestern Virginia's coal-mining industry.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Mahesh Desai, PhD, of the Luxembourg Institute of Health's Department of Infection and Immunity. Dr. Desai discusses how healthy gut bacteria may reduce the side effects of chemotherapy in cancer patients.

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.

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.