GREENWICH, Conn., September 30, 2024 – Dana’s Angels Research Trust (DART), the nonprofit organization dedicated to funding medical research, education and treatment of the rare childhood disease Niemann-Pick Type C (NPC), often referred to as “childhood Alzheimer’s,” is proud to announce that its 7th annual DART to the Finish Charity Walk, held on September 28, …
FDA Advisory Committee Votes To Recommend Arimoclomol as Effective Treatment for NPC
PDUFA Action Date for the Arimoclomol NDA is September 21, 2024 CELEBRATION, Fla., Aug. 02, 2024 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) …
FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients
FDA Press Release Link: FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients | FDA By: Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research and Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research An estimated 10,000+ rare diseases affect more than 30 million people – approximately one …