TREATMENT OF GAUCHER DISEASE USING GLUCOCEREBROSIDASE INFUSION
$0M01FY2002RRNIH
Children'S Hosp Pittsburgh/Upmc Hlth Sys, Pittsburgh PA
Investigators
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Abstract
The uptake of glucocerebrosiadase is dramatically improved by exposing mannose residues in the oligosaccharide side chain of the glycoprotein. This strategy enables enzyme infusions as a therapeutic tool. The GCRC supported clinical studies are aimed at determining the lowest doses of enzyme needed to be effective treatment.
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