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News

  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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