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Research

Alongside providing support and care for those living with or affected by Niemann-Pick disease, NPUK also provides information relating to research. NPUK shares knowledge, expertise and works in partnership and collaboration with other not-for-profit organisations, medical authorities and scientific institutions across the world, in order to facilitate progress towards therapeutic interventions for the Niemann-Pick disease.

Where possible, NPUK also funds appropriate research in order to improve medical and scientific knowledge and promote understanding of the disease. The results are often shared amongst our community of families at the annual family conference we hold every September. It is through this medical research and clinical trials that we will find innovative new ways in which to better manage Niemann-Pick disease.

Research

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Latest news

25.08.16

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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25.08.16

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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