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Niemann-Pick Disease

Niemann-Pick diseases are a group of rare 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 devastating neurodegenerative disease caused by an accumulation of lipids (fats) in the liver, brain and spleen.

Niemann-Pick Disease

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Latest news

25.08.16

NP-C Quality of Life Questionnaire – Can you Help?

You may recall that Niemann-Pick UK has been supporting Aston University in their work to deliver a research study titled “Development of health-related quality of life scales for patients and parents affected by Niemann-Pick Disease Type C”, which aims to bring benefits for the NP-C community.

This study has ethical and R&D approvals through the appropriate UK academic channels and in order to make further progress, research student Lydia needs your help.

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25.08.16

An Exciting Announcement from Vtesse

Vtesse have announced an agreement to be acquired by Sucampo Pharmaceuticals—a global biopharmaceutical company that, like Vtesse, is focused on pursuing scientific innovation with a passion for improving the lives of patients.

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