Niemann Pick disease is currently incurable. There are also only limited forms of therapy to combat the symptoms. Research activity also varies depending on the type of disease.
Type A
There is no effective treatment for type A.
Type B
Bone marrow transplants have been carried out on patients with type B, some with encouraging results. A promising enzyme replacement therapy is also being developed and tested by Sanofi.
Type C
There are various research projects for type C. However, many of them are still in the early stages. In Switzerland, the drugs "Zavesca" from Janssen and "Miglustat Dipharma" (generic) from EffRx are approved. These are intended to slow the progression of the disease. The most current information can be found at https://www.inpda.org/updates/
The medical information was provided by Dr. med. Marianne Rohrbach, Department of Metabolic Diseases, University Children's Hospital Zurich (CH).
For more information:
- Orphanet - The portal for rare diseases and Orphan Drugs
- Niemann-Pick-c.com (Actelion)
- Genetics Home Reference (U.S. National Library of Medicine, Dep. of Health and Human Services)
- University of Notre Dame (Indiana, USA)