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  1. 04.10.22

    NPUK Annual Family Conference & Interactive Workshop on Niemann-Pick Diseases 2022 [PROGRAMME]

    NPUK Chair, Will Evans: "...on behalf of the NPUK Board of Trustees, I have pleasure in welcoming you to our flagship events - the Interactive Workshop on Niemann-Pick Diseases and our Family Conference. We are pleased to be back at Wyboston Lakes; and to be able to bring our community together with once again.  As in previous years, the programme is full of interesting and valuable talks, presentations, and workshops, which will cover many thought-provoking areas of research and advances in treatments..." 

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  2. 03.10.22

    301 NPC UK Recruitment Update - IntraBio

    IntraBio Ltd. is pleased to share that the Phase III pivotal trial with N-acetyl-L-leucine (IB1001-301) clinical trial has enrolled over 30% of the target number of patients in the first month of recruitment. The study, which investigates N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC), will enroll a total of approximately 52 patients across all international sites. Since recruitment commenced in September 2022, sixteen (20) patients have been screened...

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  3. 08.09.22

    Drug Repurposing for Rare Diseases 2022 is shifting its programme to match the new drug repurposing landscape.

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  4. 19.08.22

    Families Registration for the much-loved NPUK Annual Family Conference & Interactive Workshop on Niemann-Pick Disease 2022 is now officially open!

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  5. 28.06.22

    Press Release: Xenpozyme® (olipudase alfa) approved by European Commission as first and only treatment for ASMD

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