Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Vallerie McLoughlin, MD, director of the Pulmonary Hypertension Program at the University of Michigan-Ann Arbor, about results of a phase 3 trial for ralinepag to treat pulmonary arterial hypertension.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Craig Martin, founder and CEO of the Orphan Therapeutics Accelerator (OTXL) — a nonprofit biotech dedicated to advancing shelved clinical-stage therapies for ultrarare diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Henriette Farkas, MD, PhD, director of the Hungarian Angioedema Reference Center in Budapest, and a world expert on the treatment of HAE.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Antón Blatnik, PhD, who recently won a $150,000 grant from Cure SMA to study the molecular mechanisms that drive neuromuscular disease and gene expression.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kathryn Swoboda, MD, faculty emerita at Massachusetts General Hospital and a neurologist and rare disease specialist who's been working on SMA for nearly 30 years.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dutch neurologist Ewout Groen, PhD, a member of the Scientific Advisory Board of SMA Europe.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jaime Moore, MD, about weight loss drugs—specifically the role of GLP-1 receptor agonists in treating children with neuromuscular diseases and obesity.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric psychologist Natalie Truba, PhD, on the psychological aspects of gene therapy in neuromuscular disease.

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.