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  1. 03.01.18

    Will Evans, NPUK Chair - Message for 2018

    NPUK Chair, Will Evans, gives his 2018 message...

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  2. 13.12.17

    CTD Holdings Receives Rare Pediatric Disease Designation for Trappsol® Cyclo™

    CTD Holdings, Inc. today announced that it has received rare pediatric disease designation from the FDA for its Trappsol® Cyclo™ drug...

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  3. 20.11.17

    Orphazyme’s CEO, Anders Hinsby: Letter to NP-C Community

    A letter to the NPC community from Orphazyme’s CEO, Anders Hinsby. We are please to also provide translations (from English into German, French, Spanish, Italian, Polish) of this letter.

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  4. 05.10.17

    Update #17 on the AIDNPC Clinical Programme

    The study was recently completed in May 2017. A total of 35 patients were enrolled across 12 sites in 8 countries. The primary objective of the 001 study was to characterize the individual patient’s disease progression profile through clinical, biological and quality-of-life measures recorded prospectively, as well as the historic disease information collected from patient medical records.

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  5. 20.09.17

    Route of Administration Preferences Study

    There is an opportunity for you to participate in a market research study about Niemann-Pick Type C, conducted by an organization called ApotheCom on behalf of an industry sponsor. The goal of the study is to understand your preferences and concerns regarding the method through which a drug could be administered to manage Niemann-Pick Type C.

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