International Niemann–Pick Disease Alliance

Updates

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

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  3. 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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  4. 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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  5. Orphazyme: NPC Community Notification

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  6. Mallinckrodt Reaches Agreement to Divest Experimental Drug Adrabetadex to Mandos, LLC

    Company plans to produce up to an additional two years’ supply of drug for eligible patients Agreement is subject to approval by U.S. Bankruptcy Court in Delaware Mallinckrodt Pharmaceuticals has reached an agreement to divest and transfer the Investigational...

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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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