International Niemann–Pick Disease Alliance

Updates

  1. Orphazyme provides regulatory and financial updates

    Copenhagen – October 5, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company, today announces regulatory updates for its investigational drug candidate, arimoclomol, for which it is seeking marketing authorization in both Europe and the United States for...

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  2. Cyclo Therapeutics Appoints Lise Lund Kjems, MD, PhD as Chief Medical Officer

    Sep 27, 2021 – Dr. Kjems is a leading global physician scientist with a proven pharma track record of drug development across multiple therapeutic areas, including chronic indications, oncology and rare diseases – – Gerald F. Cox, MD, PhD will...

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  3. NPC Listening Session Aug 3 Report

    We are pleased to share the following Niemann-Pick Type C Community Listening Session with the U.S. Food and Drug Administration (FDA), which was originally held on August 3rd 2021. The meeting arranged and the subsequent report prepared in a collaboration between...

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  4. Pfrieger’s Digest – 09/2021 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...

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  5. Orphazyme announces publication of results from its Phase 2/3 trial of arimoclomol in Niemann-Pick disease type C in the Journal of Inherited Metabolic Disease

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  6. IntraBio Reports Statistically Significant and Clinically Meaningful Improvements in the Use of IB1001 For Treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease)

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  7. NPC Letter to FDA (13th August 2021)

    The INPDA is committed to improving outcomes for the global Niemann-Pick community and to ensuring equitable access to safe and effective therapies.  In support of the US patient community and in recognition of the global impact of FDA decision...

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  8. INPDA Spotlight on Associazione Italiana Niemann Pick Onlus

    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...

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  9. Orphazyme presents 36-month data supporting durable response to arimoclomol during Parseghian Scientific Conference for NPC Research

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  10. INPDA Community Webinar Series: Cyclo Therapeutics – Phase 3 Trial

    Join us on Wednesday June 30th at 7pm BST to hear an update direct from Cyclo Therapeutics regarding their Phase 3 Clinical trial of Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C: The INPDA is pleased to...

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