International Niemann–Pick Disease Alliance


  1. INPDA Spotlight on NPSuisse

    Sometimes in such a big organisation as the INPDA, our various member groups can be overshadowed, but we want to show just how important each and every member group is in making the INPDA work. In the INPDA Spotlight this...

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  2. Cyclo Therapeutics Announces Last Patient Last Visit in Phase 1/2 Trial Evaluating Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C

    – Topline results from Phase 1/2 study on track for March 2021 – Individual and cumulative data seen to-date show Trappsol® Cyclo™ to be well tolerated with a favorable safety profile for all dose groups and to have encouraging...

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  3. Cyclo Therapeutics Appoints Gerald F. Cox, MD, PhD as Acting Chief Medical Officer

    – Seasoned biotechnology executive with 20-year successful track record of drug development for rare genetic diseases and extensive worldwide regulatory experience – Internationally renowned for clinical development of innovative drugs with broad experience in small molecules, enzyme replacement therapies,...

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  4. Orphazyme Appoints Christophe Bourdon as Chief Executive Officer

    Copenhagen, Denmark, March 1, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (“Orphazyme”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announced the appointment of Christophe Bourdon as the company’s Chief Executive...

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  5. Cyclo Therapeutics Unveils New Corporate Identity and Reaffirms Commitment to Improving Quality of Life and Providing Hope for Patients and Families

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  6. 17 de febrero Día Nacional de Niemann-Pick – Fundación Niemann-Pick de España

    Julia González, Coordinadora familiar, at Fundación Niemann-Pick de España provided the INPDA with the charity’s official statement for National Niemann-Pick Day, which takes place today on February 17th 2021. “…one of the ways we have to raise awareness in...

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  7. Cyclo Therapeutics Presents Positive Data from Clinical Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021

    Data from ongoing Phase 1 extension study shows improvement in disease features or disease stabilization with home-based intravenous infusions of Trappsol® Cyclo™ Phase 1 biomarker 24S-hydroxycholesterol demonstrates clearance of excess cholesterol from the brain after intravenous infusions Interim analysis...

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  8. Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium™

    Orphazyme US Investor news                                                                       ...

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  9. Adrabetadex 2021: webinar featuring Dr. Porter and Dr. Berry-Kravis discussing the Adrabetadex Program

    You can find the video recording from the Ara Perseghian Medical Research Fund’s February 3, 2021 webinar below, where Dr. Elizabeth Berry-Kravis and Dr. Denny Porter presented their Expanded Access Program data, Natural History Data, and recent longitudinal data analysis...

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  10. Cyclo Therapeutics to Present on Its Clinical and Drug Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021

    We are pleased to share the latest update from Cyclo Therapeutics entitled “Cyclo Therapeutics to Present on Its Clinical and Drug Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021”:

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