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...
by Madaline Spencer | Jun 19, 2024
Mark Frohlich, MD, CEO of Indapta Therapeutics, discusses the Phase 1 clinical trial for investigational drug candidate IDP-023, to treat patients with rare blood cancers. Non-Hodgkin lymphoma (NHL) is a group of malignant cancers originating from the...
by Madaline Spencer | Jun 18, 2024
Michelle Davis, Executive Director of the International Fibrodysplasia Ossificans Progressiva Association (IFOPA), gives an overview of this rare disease as well as IFOPA’s advocacy work. FOP is a rare disorder in which skeletal muscle and connective...
