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Niemann-Pick Disease

Niemann-Pick diseases are a group of rare and devastating inherited lysosomal storage disorders that can affect both children and adults:

Acid Sphingomyelinase Deficiency (ASMD) includes Niemann-Pick Disease type A (NP-A) and type B (NP-B), which are caused by a lack of the enzyme acid sphingomyelinase leading to a build-up of toxic materials in the body. Niemann-Pick Disease Type C (NP-C) is a hugely life-limiting neurodegenerative disease caused by an accumulation of lipids (fats) in the liver, brain and spleen.

At NPUK we are dedicated to supporting the individuals and families affected by these conditions. We offer practical and emotional support, provide information and strive to raise further awareness of the disease(s) in the hope of facilitating research and potential therapies.

Niemann-Pick Disease

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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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