Thank you for donating!

You can donate using the following services.

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

Support us

Latest news

20.11.17

Orphazyme’s CEO, Anders Hinsby: Letter to NP-C Community

A letter to the NPC community from Orphazyme’s CEO, Anders Hinsby. We are please to also provide translations (from English into German, French, Spanish, Italian, Polish) of this letter.

Read more

05.10.17

Update #17 on the AIDNPC Clinical Programme

The study was recently completed in May 2017. A total of 35 patients were enrolled across 12 sites in 8 countries. The primary objective of the 001 study was to characterize the individual patient’s disease progression profile through clinical, biological and quality-of-life measures recorded prospectively, as well as the historic disease information collected from patient medical records.

Read more
View all news Subscribe