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 …
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Zevra Announces FDA Advisory Committee Meeting To Review Arimoclomol For The Treatment Of NPC
July 9, 2024 at 9:00 AM EDT Meeting with the recently formed GeMDAC set for August 2, 2024 CELEBRATION, Fla., July 09, 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) has indicated that it will convene a meeting with the recently formed Genetic …
Azafaros announces positive topline Phase 2 study data with nizubaglustat in NPC and GM2
16 July 2024 Leiden, The Netherlands, 16 July 2024 – Azafaros B.V. today announced positive topline data from its RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat in patients with a genetic diagnosis of either GM2 gangliosidosis or Niemann-Pick disease type C (NPC). About the Trial The trial was conducted across three sites in Brazil, …