International Niemann–Pick Disease Alliance

Updates

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

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

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  5. 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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  6. IntraBio Announces Positive Pivotal Trial Results of IB1001 for the Treatment of Niemann-Pick Disease Type C

    – Phase 3 trial met the primary endpoint and key secondary endpoints showing high statistical significance – IB1001 showed a clinically meaningful improvement in symptoms, functioning, quality of life, and cognition in both pediatric and adult patients with NPC...

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  7. Cyclo Therapeutics – Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients

     

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  8. Open Letter for NPC Community [Bloomsbury Genetic Therapies Limited]

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  9. Cyclo Therapeutics – Phase 3 Study of NPC now Enrolling in UK

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  10. Pfrieger’s Digest – 8th Issue

    “Pfrieger’s Digest” is written by Frank Pfrieger of Niemann-Pick Selbsthiifegruppe (Germany) to provide an overview of the latest advances in Niemann-Pick disease based on recent scientific publications. Frank Pfrieger is group leader at the Institute of Cellular and Integrative Neurosciences in...

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The Impact of NPC

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