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  1. 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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  2. 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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  3. 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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  4. 30.11.16

    Vtesse Receives Rare Pediatric Disease Designation by the FDA for VTS-270

    Vtesse announced early in 2016 that the FDA granted Breakthrough Therapy designation status for VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial in NPC. Both the FDA and the European Medicines Agency (EMA) previously granted Orphan Drug status to VTS-270, and the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) granted it a Promising Innovative Medicine (PIM) designation.

    "We are incredibly grateful to receive Rare Pediatric Disease designation status from the FDA for VTS-270, in addition to the Breakthrough Therapy designation status, Orphan Drug designations, and the MHRA PIM designation. These designations are granted to drugs with high promise that may address areas of unmet medical need or for rare diseases. These designations will aid us as we seek to develop VTS-270 as an effective treatment to slow the progress of NPC or stabilize patients who suffer from the devastating impacts of NPC," said Ben Machielse, Drs., President and Chief Executive Officer of Vtesse. "We recognize the considerable collaboration and efforts to advance the clinical development and regulatory processes in the rare disease space, and, above all, we continue to thank all of the parents who have supported the development of VTS-270."

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  5. 24.11.16

    Vtesse October 2016 Newsletter: Update on the Clinical Trial of VTS-270: The NPC Study

    Here is the October 2016 edition of the Vtesse Newsletter, which highlights the most recent updates of the clinical trial of VTS-270. We have made it available in a number of different languages for our global community, including; Arabic, Dutch, English, French, German, Italian, Mandarin, Spanish EU, Spanish Lat. Am, and Turkish.

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