ENZYME THERAPY FOR NEUROLOGICALLY AFFECTED PATIENTS WITH GAUCHER DISEASE

$0M01FY2000RRNIH

Children'S Hosp Pittsburgh/Upmc Hlth Sys, Pittsburgh PA

Investigators

Linked publications & trials

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Abstract

This is a long term study of patients with the clinical phenotype and genotype of neuronopathic Gaucher disease and their responses to therapy with a biotherapeutic agent. This human enzyme, macrophage-targeted glucocerebrosidase, produced under the trade name Cerezyme benefits patients without neurologic disease. It may prevent the onset of progression of neurologic disease.

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