International Niemann–Pick Disease Alliance

Updates

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

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  2. 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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  3. 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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  4. 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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  5. 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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  6. Cyclo Therapeutics, Inc. and Applied Molecular Transport Inc. Enter into a Definitive Merger Agreement

    Combined company will operate as Cyclo Therapeutics with primary focus on advancing Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 (NPC1) At closing combined Company will have approximately $13.7 million cash on hand to fund operations into 2H...

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  7. Advancing Rare Disease Research: Zevra’s Acquisition of Acer Therapeutics

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  8. Shining a Spotlight on Superstar Siblings: The Unsung Heroes of NPD Awareness Month

    Celebrating Strength and Support during NPD Awareness Month… …let’s shine a bright light on the unsung heroes of our Niemann-Pick Disease (NPD) community this October, during Niemann-Pick Disease Awareness Month. We’re thrilled to introduce our “Sibling Spotlight” campaign, dedicated...

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  9. Community Statement from Kisbee Therapeutics [30th August 2023]

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  10. Cyclo Therapeutics Announces Publication of Positive Data from Phase 2 Clinical Study of Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C1

    – Data published in official journal of Molecular Genetics and Metabolism Reports: Evidence of a change in cholesterol homeostasis in peripheral organs and the brain after the initial infusion Trappsol® Cyclo™ was detected in the CSF Completers had an 88.9% improvement...

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The Impact of NPC

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