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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

05.01.21

Cyclo Therapeutics Announces Positive Efficacy Data from Extension Protocol with Trappsol® Cyclo™ in Patients with Niemann-Pick Disease Type C

Home-based intravenous infusions of Trappsol® Cyclo™ for up to one year show improvement in disease features or disease stabilization...

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05.01.21

NPUK 2020 Support Fund

In recognition that many in our community are being impacted by the events of 2020, Trustees of NPUK and The Hollie Foundation, in recognition of their shared goals, have designated funds to create a new grant scheme for patients and families. The aim of this fund is to assist those experiencing hardship or unexpected expense, which threatens to impact their physical or mental well-being...

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