The Niemann Pick disease is not curable at present. Also, there are only limited forms of therapy to combat the symptoms.
There is no effective treatment for type A.
There are some experiments with bone marrow transplantation, partly with encouraging results. In addition, an enzyme substitute therapy is also tried out.
For the type C, there are various research projects. Most of them are still in the initial stages. In Switzerland the drug "Zavesca" is admitted. Zavesca should slow down the course of the disease.
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)
Medical information on this website is maintained by Matthias Oetterli (journalist BR and father of an NPC patient). Information is checked by Dr. med . Marianne Rohrbach, Department of Metabolic Diseases, University Children's Hospital Zurich (CH).