Gaucher Disease
Alternate Names : Glucosylceramide Storage Disease, GSDI
 See all Pictures & Images
Gaucher Disease Treatment
In the past the only potential treatment was removal of the spleen (splenectomy). This has given way to injections of a replacement synthetic enzyme (Cerezyme/Ceredase). Gene therapy is an experimental approach.
A novel oral treatment has recently been evaluated. This drug is known as N-butyldeoxynojirimycin (OGT 918). The mechanism of action is by inhibiting the formation of glucocerebroside. This trial improved key clinical features of Gaucher's disease including liver and spleen size and, to a lesser degree, blood counts. The most frequent side effect was diarrhea. This is currently being further evaluated.
|
