223: An Interview With Dr. Antón Blatnik on the Molecular Mechanisms Driving Neuromuscular Disease

227: An Interview With Dr. Andrew Wilson, Scientific Director of the Alpha-1 Foundation

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Andrew Wilson, MD, scientific director of the Alpha-1 Foundation, about the latest therapies to treat alpha-1 antitrypsin deficiency, an incurable genetic disease that causes both lung and liver damage.

226: An Interview With Dr. Vallerie McLoughlin, Director of the University of Michigan's Pulmonary Hypertension Program

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.

225: An Interview With Craig Martin, Founder and CEO of the Orphan Therapeutics Accelerator

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.

224: An Interview With Henriette Farkas, Director of the Hungarian Angioedema Reference Center in Budapest

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.

223: An Interview With Dr. Antón Blatnik on the Molecular Mechanisms Driving Neuromuscular Disease

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.