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Research

Alongside providing support and care for those living with or affected by Niemann-Pick disease, one of our other main areas of focus is providing 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 and health professionals at the Annual Family Conference & Interactive Workshop 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

14.10.19

Study VTS301 – Study Resumption in France

Study Title: A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease...

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13.09.19

CTD Holdings Announces FDA Approval of US Expanded Access Treatment Program in Niemann-Pick Disease Type C

ALACHUA, FL–(Businesswire)–13 September 2019–CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that the company will provide Trappsol® Cyclo™, its proprietary hydroxypropyl beta cyclodextrin drug, to a pediatric patient diagnosed with Niemann-Pick Disease Type C...

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