Glucocerebrosidase clinical effectiveness

Medicine in New York using glucocerebrosidase that had been produced lecont-binantly in Chinese hamster ovary cells. The enzyme obtained from these cells was modified by exposure to the- same these cxogtycosidases used previously to produce mannose-terminal placental glucoceiebrosidase (Kg, 2). The clinical effectiveness of the recombinant macrophage-targeted enzyme was found to be... 

The clinical effects of mannose-terminal glucocerebrosidase were initially examined in eight patients with non-neuronopathic, type 1 Gaucher disease. Seven of the patients were either adolescents or adults, and one was a 4-year-old boy. A total dose of 190 U of enzyme was administered intravenously to each of these patients weekly over a 6-month period. A beneficial effect was clearly evident only in the youngest of the recipients [26]. A dramatic increase in... 

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