by Madaline Spencer and James Radke, PhD | Jul 5, 2023
Brad Chapman, Head of US Epilepsy and Rare Syndromes at UCB, discusses the recent decision by the US Drug Enforcement Agency (DEA) to deschedule fenfluramine. In 2020, the FDA approved Fenfluramine as a serotonin agonist for the treatment of seizures caused by...
by Madaline Spencer and James Radke, PhD | Jun 29, 2023
Nicholas E. Johnson, MD, Associate Professor in the Department of Neurology at Virginia Commonwealth University, summarizes data from the phase 1/2 MARINA trial. This trial tested AOC 1001 in patients with myotonic dystrophy type 1 (DM1). DM1 is a progressive...
by James Radke, PhD and Madaline Spencer | Jun 14, 2023
Dean Suhr, of the MLD Foundation, provides an overview of the philosophy of their foundation when it comes to supporting research for metachromatic leukodystrophy (MLD). MLD is a genetic, lysosomal disorder caused by a deficiency in the enzyme arylsulfatase A...
by Madaline Spencer and James Radke, PhD | Jun 13, 2023
Rennie McCarthy, Chief Executive Officer at Stealth Biotherapeutics, discusses clinical trial development challenges for ultra-rare diseases. Stealth Biotherapeutics is developing elamipretide, a mitochondrial protective agent, as a possible treatment...
by Madaline Spencer and James Radke, PhD | Jun 13, 2023
Lisa Forbes Satter, MD, Associate Professor of Pediatrics at Baylor College of Medicine, explains current treatment options for patients with primary Immunodeficiency (PI). PI is a heterogeneous group of inherited disorders characterized by impaired immune...
