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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

04.10.18

CTD announces encouraging top-line data from two ongoing clinical trials of Trappsol® Cyclo™ administered intravenously in Niemann Pick Disease Type C (NPC)

CTD announces encouraging top-line data from two ongoing clinical trials of Trappsol® Cyclo™ administered intravenously in Niemann Pick Disease Type C (NPC)

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03.10.18

Orphazyme Reports Encouraging Arimoclomol Clinical Trial Top-line Data in Niemann-Pick Disease Type C (NP-C)

ORPHAZYME REPORTS ENCOURAGING ARIMOCLOMOL CLINICAL TRIAL TOP-LINE DATA IN NIEMANN-PICK DISEASE TYPE C (NPC)...

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