TREATMENT OF GAUCHER DISEASE USING GLUCOCEREBROSIDASES INFUSIONS

$3,034M01FY2000RRNIH

University Of Pittsburgh At Pittsburgh, Pittsburgh PA

Investigators

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Abstract

Gaucher disease is an inherited disorder of the lysosome caused by mutations in the gene for glucocerebrosidase. These mutations result in a deficiency of the enzyme activity. The naturally occurring substrate of the enzyme is a poorly water soluble lipid. The disease state results because of lack of glucosylcer-amide. A commercial product, approved by the FDA, is Ceredase. This study is evaluating the rate and extent of clinical response to repeated infusions in patients with Gaucher's Disease.

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