by Madaline Spencer and James Radke, PhD | Jul 10, 2023
Frank Rivera of Stronger Than Sarcoidosis and Sarcoidosis of Long Island, two patient advocacy groups focused on raising awareness and research, discusses the rare disease. Sarcoidosis is a rare, systemic, inflammatory disorder characterized by the...
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 Peter Ciszewski | Jul 4, 2023
Kim Moran, Ph.D., MBA, Head of US Rare Diseases at UCB, discusses the two orphan drugs, rozanolixizumab and zilucoplan, which the company hopes to have approved shortly to treat myasthenia gravis. Myasthenia gravis is a rare autoimmune disorder that targets the...
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 Madaline Spencer | Jun 19, 2023
Staci Kallish, DO, a medical geneticist at Penn Medicine explains the difference between a geneticist and a genetic counselor. A geneticist is a physician trained in the identification and management of genetic conditions, either through genetic residency...
