What is the treatment for niemann-pick disease?

N-P Disease. Treatments for niemann–pick disease are limited with care being mostly supportive. Anecdotally, organ transplant has been attempted with limited success. Future prospects include enzyme replacement and gene therapy. Bone marrow transplant has been attempted for type b. Supportive care through nutrition, medication, physical therapy and being followed by specialists can help with quality of life.

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Are there treatments for niemann pick disease?

Clinical trials. Niemann pick is a progressive disease due to metabolic abnormality that leads to the improper degradation of lipids and build-up in the lysosomes. There are different forms. In general, there is no cure at this time. There are clinical trials with medications and bone marrow transplants that have some promise. Nih has some labs directed at basic and clinical research for niemann pick. Read more...