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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

11.06.19

Orphazyme to prepare for filing of arimoclomol in Europe for Niemann-Pick disease Type C (NPC)

Orphazyme plans to meet with the US Food and Drug Administration (FDA) to discuss the pathway forward during the Summer and will provide an update after it has received written comments from the FDA...

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03.06.19

CTD Holdings Closes $7.4 Million Private Placement

CTD Holdings, Inc. (OTCQB: CTDH)("CTD" or the "Company"), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that it closed its previously announced private placement of securities with a group of institutional and accredited investors that included directors and officers of the Company.

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