by Madaline Spencer | Jun 26, 2024
Alan Krasner, MD, Chief Endocrinologist at Crinetics Pharmaceuticals, discusses a series of trials testing the safety and efficacy of investigational drug paltusotine for the treatment of acromegaly. Acromegaly is a rare endocrine disorder resulting from...
by Madaline Spencer | Jun 25, 2024
Marie Abrego, a person with neuromyelitis optica spectrum disorder (NMOSD), and Wendy Erler, Vice President of Patient Experience at Alexion Pharmaceuticals Inc, discuss getting diagnosed with NMOSD and finding support. NMOSD is a rare autoimmune...
by Madaline Spencer | Jun 24, 2024
Greg Josephs, Myasthenia Gravis Foundation of America (MGFA) Volunteer Ambassador and patient with myasthenia gravis (MG), discusses living with a rare disease and valuable resources available to patients. MG is a rare chronic autoimmune neuromuscular...
by Madaline Spencer | Jun 21, 2024
Leslie Edwin, President of the Cushing’s Support and Research Foundation (CSRF), and Gretchen Jordan, Associate Director of the CSRF, discuss how their organization supports patients with Cushing’s disease/syndrome. Cushing’s syndrome is a rare...
by Madaline Spencer | Jun 20, 2024
Lawrence Lustig, MD, Otolaryngology-Head & Neck Surgery at Columbia University, discusses preliminary results from the CHORD trial testing the safety, efficacy, and tolerability of investigational gene therapy, DB-OTO, in children with otoferlin-related auditory...
