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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

24.05.19

CTD Announces Home-Based Dosing of First Patient in US Extension Protocol of Clinical Trial using Intravenous Administration of Trappsol® Cyclo™ for Niemann-Pick Disease Type C

ALACHUA, FL – (Accesswire) – May 23, 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 its first home-based infusion of its proprietary formulation of hydroxypropyl beta cyclodextrin, Trappsol® Cyclo™, in a formal clinical trial. The trial is “An Open-Label Extension Study of the Long-Term Safety and Efficacy of Intravenous Trappsol® Cyclo™ (HPBCD) in Patients with Niemann-Pick Disease Type C (NPC-1)” (ClinicalTrials.gov NCT03893071) in the United States.

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13.05.19

Professional Delegate Interactive Workshop and Conference Registration Form - 2019

NPUK INTERACTIVE WORKSHOP - REGISTER NOW! For all health professionals, clinicians and pharma reps who want to join us for the Interactive Workshop this form is for you, please register by the 6th September.

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