Article
Cessation of enzyme replacement therapy in Gaucher disease.
Genetics in medicine : official journal of the American College of Medical Genetics - 1 Jan 2000
Grinzaid Karen A, Geller Eleanor, Hanna Soheil L, Elsas Louis J
Abstract excerpt
PURPOSE: Enzyme replacement therapy (ERT) is a promising therapeutic intervention for lysosomal storage diseases. Posttranslationally engineered human beta-glucocerebrosidase (Ceredase/Cerezyme) is commercially available and is the standard ERT for Type I Gaucher disease. Cessation of therapy is sometimes necessary for personal or financial reasons, but the consequences of discontinuation are unknown. This study...
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