International Niemann–Pick Disease Alliance

Updates

  1. Pfrieger’s Digest – 01/2020 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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  2. Orphazyme Announcement: US Early Access Program for NPC

    Copenhagen, Denmark, January 6, 2020 – Orphazyme A/S (ORPHA.CO), a biopharmaceutical company pioneering Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces the availability of an Early Access Program (EAP) in the United States (US) for its...

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  3. Orphazyme reports positive arimoclomol data from open-label phase 2/3 extension in Niemann-Pick disease Type C

    • 12-month data from open-label extension trial show sustained effect in reducing disease progression over two years • New genetic sub-group analysis recommended by US Food and Drug Administration supports robustness of results • Filling of New Drug Application...

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  4. Expanded Access Programme (EAP) – An Update from Orphazyme

    Today, the INPDA received the attached letter (copied below), from Regan Sherman, Associate Director, Patient Advocacy Relations at Orphazyme.  The letter provides the latest update regarding the introduction of an EAP programme for their investigational therapy, arimoclomol.  Please do...

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  5. WELL DONE: Cláubia Viegas Bender

    This week Cláubia Viegas Bender, co-founder of INPDA member group Niemann-Pick B-RS, completed her master’s degree with a project focused on ASMD Niemann-Pick disease type B. We are all hugely proud of her achievement, and we hope you will join...

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  6. Cyclo Therapeutics, Inc. to Present Clinical Trial Data on Niemann-Pick type C Disease at the 16th Annual WORLDSymposium

    Data show that Trappsol® Cyclo™, the Company’s proprietary hydroxypropyl beta cyclodextrin drug, reduces levels of trapped cholesterol in liver tissue of Niemann-Pick Disease Type C Patients GAINESVILLE, FL – (Businesswire) – December 16, 2019 – Cyclo Therapeutics, Inc. (OTCQB:...

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  7. Orphazyme receives Breakthrough Therapy Designation for arimoclomol in Niemann-Pick Disease Type C (NPC)

    Orphazyme receives Breakthrough Therapy Designation for arimoclomol in Niemann-Pick Disease Type C (NPC) • There are currently no US FDA-approved products for NPC • Orphazyme plans to file arimoclomol in the US in H1 2020 Copenhagen, Denmark, November 19,...

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  8. Cyclo Therapeutics Announces Its Publication of the Most Extensive Set of Case Study Reports To-date on Expanded Access Use of Hydroxypropyl Beta Cyclodextrin in Niemann-Pick Disease Type C

    Publication describes favorable safety profile and efficacy in neurologic and other disease features in individual NPC patients following intravenous administration of drug Gainesville, FL – (Businesswire) – 31 October 2019 – Cyclo Therapeutics, Inc, (OTCQB: CTDH), a clinical stage...

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  9. Cyclo Therapeutics Announces Completion of Enrollment in its Phase I Trial to Evaluate Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C

    Top line results are expected to be available in February 2020 GAINESVILLE, FL – (Businesswire) – October 22, 2019 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), formerly CTD Holdings, Inc., a biotechnology company that develops cyclodextrin-based products for the treatment...

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  10. Cyclo Therapeutics Announces Its Support for The Firefly Fund’s Newborn Screening Program

    Announcement made in conjunction with October 19th Global Awareness Day for Niemann-Pick Disease sponsored by International Niemann Pick Disease Alliance GAINESVILLE, FL – (Businesswire) – October 18, 2019 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), formerly CTD Holdings, Inc., a...

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