Niemann-Pick
Alternate Names : Sphingomyelinase Deficiency (Type A Niemann-Pick Disease)
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Niemann-Pick Treatment
For Types A and B Niemann-Pick the ASM gene has been isolated and extensively studied. DNA testing and prenatal diagnosis is currently available.
Research into treatments for Types A and B NPD has progressed rapidly since the early 1990's. Mount Sinai School of Medicine is conducting research on bone marrow transplantation, enzyme replacement therapy, and gene therapy. All of these therapies have had some success against Type B Niemann-Pick in a laboratory environment. Unfortunately, none of the potential therapies has been effective against Type A.
For Type C Niemann-Pick, no specific treatment is available. A healthy, low-cholesterol diet is recommended. However research into low-cholesterol diets and cholesterol-lowering drugs do not indicate that these halt the progress of the disease or change cholesterol metabolism at the cellular level. Additionally, many Type C symptoms, such as cataplexy and seizures, can be controlled or tempered by drugs.
The National Niemann-Pick Disease Foundation website has more in-depth reporting on the research into potential treatments.
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