International Niemann–Pick Disease Alliance

Updates

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

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  2. 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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  3. 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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  4. Zevra Acer Deal Close [INPDA NPC PAG Letter]

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  5. Pfrieger’s Digest – 9th Issue

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  6. Introducing Neil F. McFarlane: Zevra Therapeutics’ New President and CEO

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  7. Sibling Spotlight: Alejandro

    Niemann-Pick Disease Awareness Month is the perfect time to acknowledge the invaluable role that siblings play in providing support and love to their affected family members. Cuando Alejandro tenía 2 añitos, llegó al mundo su hermana tan esperada, Natalia....

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  8. Sibling Spotlight: Sara

    As part of this year’s Niemann-Pick Disease Awareness Month, we are putting the spotlight on siblings as we feel they are often a huge support to both their brother or sister, as well as the broader family. They are also some...

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  9. Sibling Spotlight: Iker

    As part of this year’s Niemann-Pick Disease Awareness Month, we are putting the spotlight on siblings as we feel they are often a huge support to both their brother or sister, as well as the broader family. They are also some...

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  10. Sibling Spotlight: Guillermo

    As part of this year’s Niemann-Pick Disease Awareness Month, we are putting the spotlight on siblings as we feel they are often a huge support to both their brother or sister, as well as the broader family. They are also some...

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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...

The Impact of NPC

The global community tell their stories...