International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. 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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  3. Cyclo Therapeutics – Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients

     

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

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

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  6. Pfrieger’s Digest – 03/2023 Edition

    “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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  7. Pfrieger’s Digest – 09/2022 Edition

    “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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  8. Azafaros Receives FDA’s IND Clearance and Fast Track Designation for Lead Asset AZ-3102

    Leiden, The Netherlands, January 5, 2023 – Azafaros B.V. today announced that it has received Investigational New Drug (IND) clearance from the United States Food and Drug Administration (FDA) to conduct a clinical Phase 2 trial for its lead...

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  9. IB1001-301 Recruitment Complete

    IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 – NCT05163288). Enrolment for IB1001-301 commenced...

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  10. Cyclo Therapeutics Announces Publication of Phase 1 Data for Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C1 (NPC1)

    – Data published in official journal of the Society for Inherited Metabolic Disorders, Molecular Genetics and Metabolism – Published trial data show that Trappsol® Cyclo™ overcomes the NPC1 defect by removing trapped cholesterol from cells both systemically and in the...

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