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News

  1. 28.02.17

    Orphazyme: Update #14 on the AIDNPC Clinical Programme

    'With the AIDNPC study close to being fully recruited, Orphazyme proposed a change of frequency for the now-monthly teleconference calls providing updates. Once the last patient has been enrolled, the updates could be provided on a quarterly basis. The company invites the patient organisations to share their views on the proposed change in TC frequency.'

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  2. 10.02.17

    Vtesse January 2017 Newsletter: Update on the Clinical Trial of VTS-270: The NPC Study

    On 28 February 2017, the tenth edition of Rare Disease Day will see thousands of people from all over the world come together to advocate for more research on rare diseases. Over the last few decades, funds dedicated to rare disease research have increased. But it can’t stop there.

    Rare Disease Day 2017 is therefore an opportunity to call upon researchers, universities, students, companies, policy makers and clinicians to do more research and to make them aware of the importance of research for the rare disease community.

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  3. 30.01.17

    Happy 2nd Birthday, Aparito!

    Niemann-Pick UK and the International Niemann Pick Disease Alliance would like to congratulate Aparito on celebrating two successful years of working in partnership with patient organisations, hospitals and research institutions across the World!

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  4. 17.01.17

    CTD Holdings Receives FDA Fast Track Designation for Trappsol® Cyclo™

    The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Trappsol® Cyclo™ for the treatment of systemic Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease. Trappsol® Cyclo™ is the Company's proprietary formulation of hydroxypropyl beta cyclodextrin in development for the treatment of NPC; it has previously been designated an Orphan Drug by the FDA and the European Medicines Agency.

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  5. 04.01.17

    Jim Green: Stepping Down as NPUK Trustee

    Jim Green, a founding member of NPUK all the way back in 1991 - and therefore an individual who has been connected to Niemann-Pick UK for over 25 years, is stepping down from the charity's Board of Trustees. ..

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