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Correspondence
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Volume 339:1165-1166 October 15, 1998 Number 16
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Cardiac Response to Enzyme-Replacement Therapy in Gaucher's Disease

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To the Editor: Gaucher's disease is the most common lysosomal-storage disorder. It is caused by a deficiency of glucocerebrosidase, which results in an accumulation of glucosylceramide in abdominal organs and bone, sometimes in the nervous system, and in rare cases, in the heart. We describe a patient with Gaucher's disease involving the heart in whom therapy with glucocerebroside (alglucerase) was beneficial.

The patient was found to have Gaucher's disease at the age of six years, at which time splenectomy was performed because of massive splenomegaly and thrombocytopenia. She subsequently had progressive hepatomegaly and bone pain. At the age of 16 . . . [Full Text of this Article]

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