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Niemann-Pick Type C Therapy Accelerator
Niemann-Pick Type C Therapy Accelerator
University of Notre Dame

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Home 2020 September

NTA to work with Companies & NIH to have the FDA evaluate Miglustat as an indication for NPC disease

National Institutes of Health

Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease.

Recent Posts

  • NPC Listening Session with the FDA on August 3rd – Final Report
  • Pfreigers digest September 2021
  • Letter from the NPC community following the FDA Listening Session on August 3, 2021
  • press release from Orphazyme concerning Arimoclomol
  • Notes from NPC Accelerator Meeting – 5-26-21

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    • Our Story
      • About the NPC Therapy Accelerator
      • Oversight Committee
    • What is NPC Disease?
    • Research Collaboration
      • Clinical Hospitals
      • Community Initiatives
      • Federal Agencies
      • NPC Research Tools
      • Researchers and Programs
      • Trending Research
    • Therapies
      • _
      • Companies Developing NPC Therapies
      • Get Involved in a Clinical Trial
      • Therapies in Development
    • Get Involved
      • Foundations, Organizations & Conferences
      • Funding Opportunities
    • Contact