International Niemann–Pick Disease Alliance

Updates

  1. Azafaros announces positive topline Phase 2 study data with nizubaglustat

    Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C. Study primary endpoint met, with confirmed safety profile Investigators and Patient Organisations supportive of continued development Leiden, The Netherlands, 19 June...

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  2. Azafaros: Completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients

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  3. Pfrieger’s Digest – 02/2024 Edition

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  4. Zevra Therapeutics: FDA Extends Review Period for Arimoclomol NDA

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  5. IntraBio: NEJM Publishes Expert Perspective Editoral on IB1001

    IntraBio Inc is pleased to announce that The New England Journal of Medicine (NEJM) has published a Science Behind the Study Expert Perspective Editorial: N-Acetyl-l-Leucine and Neurodegenerative Disease. The Editorial reports on the broad potential of N-acetyl-L-leucine (IB1001) as...

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  6. NEJM publishes IB1001-301 Trial Results

    IntraBio Inc is pleased to announce that The New England Journal of Medicine (NEJM) has published the detailed results of the Phase 3, Pivotal Trial with N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC). New England...

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  7. Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled

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  8. Cyclo Therapeutics Announces Positive Outcome from Type C Meeting with the U.S. FDA Discussing Trappsol® Cyclo™ Clinical Program for the Treatment of Niemann-Pick Disease Type C1

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  9. Introducing the INPDA International Niemann-Pick Disease Alliance Executive Committee

    Introducing the newly elected INPDA International Niemann-Pick Disease Alliance Executive Committee (from left to right); Sarah Janetta, Executive Communications Officer, Joslyn Crowe, Vice President, Sandy Cowie, President and Toni Mathieson, Executive Secretary. We are so proud of the team we have...

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  10. Zevra Acer Deal Close [INPDA NPC PAG Letter]

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