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  1. 17.01.20

    IntraBio Neuroprotection and Disease Modification for Niemann-Pick C Extension Phase Approved in Europe

    OXFORD, UK / ACCESSWIRE / January 15, 2020 / IntraBio Inc., a late-stage biopharmaceutical company, is pleased to share that the Extension Phase for the IB1001-201 Clinical Trial has been approved in all European countries where the trial is being conducted...

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  2. 06.01.20

    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 investigational drug arimoclomol for the treatment of Niemann-Pick disease Type C (NPC)...

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  3. 03.01.20

    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 in US on track for H1 2020, with expected approval in H1 2021

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  4. 19.12.19

    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...

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  5. 29.11.19


    We are joining 140+ organisations in a call to all political parties to put children and young people at the heart of this General Election...

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