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News

  1. 13.04.17

    NP-C Quality of Life Questionnaire – Can you Help?

    You may recall that Niemann-Pick UK has been supporting Aston University in their work to deliver a research study titled “Development of health-related quality of life scales for patients and parents affected by Niemann-Pick Disease Type C”, which aims to bring benefits for the NP-C community.

    This study has ethical and R&D approvals through the appropriate UK academic channels and in order to make further progress, research student Lydia needs your help.

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  2. 05.04.17

    An Exciting Announcement from Vtesse

    Vtesse have announced an agreement to be acquired by Sucampo Pharmaceuticals—a global biopharmaceutical company that, like Vtesse, is focused on pursuing scientific innovation with a passion for improving the lives of patients.

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  3. 23.03.17

    WE NEED YOUR HELP: Westminster Hall Debate

    WE NEED YOUR HELP: Just last week, NICE announced its decision to implement an upper funding threshold for its Highly Specialised Technology Programme, the main route used to evaluate medicines for rare disease patients. If implemented, the plans will significantly affect rare disease patients and their ability to access life-changing treatment, delivered at a time when we should be expanding access routes rather than further limiting them.

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  4. 21.03.17

    CTD Holdings Initiates Recruitment for Phase I/II Clinical Study in Europe of Trappsol® Cyclo™ for Treatment of Niemann-Pick Disease Type C

    “We are pleased to be participating in this promising clinical trial,” said Dr. Sharma. “Based on the compelling compassionate use data generated to date, we believe that Trappsol® Cyclo™ administered intravenously has the potential to effectively treat NPC, for which a significant unmet need exists, with only one approved treatment in Europe and none in the U.S. We look forward to evaluating Trappsol® Cyclo™ in the clinical setting in order confirm these encouraging results.”

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  5. 16.03.17

    Vtesse Completes Enrollment of Phase 2b/3 Pivotal Clinical Trial of VTS-270 in NP-C

    "Completing enrollment in the pivotal Phase 2b/3 clinical trial of VTS-270 is a significant milestone for the NPC community and Vtesse, and we are fortunate to have exceeded our goal of 51 participants. We are extremely grateful to the study participants and their families, as well as the clinical site investigators and staff, for their collective commitment and significant contributions to the development of a potential treatment for NPC," said Ben Machielse, Drs., Vtesse's President and Chief Executive Officer. "We look forward to generating topline results from the study early next year."

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