International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. 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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  3. Orphazyme presents 36-month data supporting durable response to arimoclomol during Parseghian Scientific Conference for NPC Research

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  4. 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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  5. Orphazyme provides regulatory update from FDA on arimoclomol for Niemann-Pick disease type C

    Orphazyme A/S Company announcement No. 16/2021 Inside information Company Registration No. 32266355 Copenhagen – June 18, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases, today...

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  6. Orphazyme: NPC Community Notification

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  7. Cyclo Therapeutics Appoints Lori McKenna Gorski as Global Head of Patient Advocacy

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  8. INPDA Spotlight on Fundación Niemann-Pick de España (FNP)

    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. Cyclo Therapeutics Announces Design of Pivotal Phase 3 Study Evaluating Trappsol® Cyclo™ in Niemann-Pick Type C1

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  10. Cyclo Therapeutics Inc: Update (13/4/21)

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

The Impact of NPC

The global community tell their stories...