FDA Approves First Treatment for Niemann-Pick Disease, Type C | FDA For Immediate Release:September 20, 2024 Today, the U.S. Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in …
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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 …
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, …