International Niemann–Pick Disease Alliance

Updates

  1. INPDA Spotlight on VML France Niemann Pick Group

    Sometimes in such a big organisation as the INPDA, our various member groups can be overshadowed, but we want to show just how important each and every member group is in making the INPDA work. In the INPDA Spotlight this...

    Read story
  2. Pfrieger’s Digest – 03/2021 Edition

    “Pfrieger’s Digest” is written by Frank Pfrieger of Niemann-Pick Selbsthiifegruppe (Germany) to provide an overview of the latest advances in Niemann-Pick disease based on recent scientific publications. Frank Pfrieger is group leader at the Institute of Cellular and Integrative Neurosciences in...

    Read story
  3. Cyclo Therapeutics Meets Primary Efficacy Endpoint in Phase 1/2 Trial from Intravenous Trappsol® Cyclo™ in Rare Disease Niemann-Pick Type C1 (NPC1)

    100% of patients who completed the trial improved or remained stable, and 89% met the efficacy outcome measure of improvement in at least 2 domains of the 17-domain NPC Severity Scale Trial data suggest that Trappsol® Cyclo™ overcomes the...

    Read story
  4. University College London begins preclinical studies to develop a treatment for Niemann Pick C disease with support from the Columbus Foundation

    Read story
  5. Cyclo Therapeutics to Participate in Inaugural Emerging Growth Virtual Conference

    – Presenting Innovative Small and Mid-Cap Companies in Healthcare, Industrials, Electric Vehicles, TMT, and Consumer Products Gainesville, FL – (Business wire) – March 12, 2021 — Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage...

    Read story
  6. Cyclo Therapeutics Appoints Russ Belden as Acting Chief Commercial Officer

    – Preeminent biotechnology commercialization leader with over 33 years of industry experience, including 16 years at Genentech with a pivotal role in commercializing products across multiple therapeutic areas Gainesville, FL – (Business wire) – March 10, 2021 — Cyclo...

    Read story
  7. Cyclo Therapeutics Receives Positive Opinion from the Paediatric Committee of the European Medicines Agency on the Agreement of a Paediatric Investigation Plan for Trappsol® Cyclo™

    Gainesville, FL – (Business wire) – March 9, 2021 — Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families suffering...

    Read story
  8. INPDA Spotlight on NPSuisse

    Sometimes in such a big organisation as the INPDA, our various member groups can be overshadowed, but we want to show just how important each and every member group is in making the INPDA work. In the INPDA Spotlight this...

    Read story
  9. Cyclo Therapeutics Announces Last Patient Last Visit in Phase 1/2 Trial Evaluating Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C

    – Topline results from Phase 1/2 study on track for March 2021 – Individual and cumulative data seen to-date show Trappsol® Cyclo™ to be well tolerated with a favorable safety profile for all dose groups and to have encouraging...

    Read story
  10. Cyclo Therapeutics Appoints Gerald F. Cox, MD, PhD as Acting Chief Medical Officer

    – Seasoned biotechnology executive with 20-year successful track record of drug development for rare genetic diseases and extensive worldwide regulatory experience – Internationally renowned for clinical development of innovative drugs with broad experience in small molecules, enzyme replacement therapies,...

    Read story

Join the Registry

Calling all patients and carers! We've created a global registry of all NPD patients - if you're not enrolled yet, join us now...

Think Again. Think NP-C

Join our international campaign to improve diagnosis of NP-C!