ENZYME REPLACEMENT THERAPY IN GAUCHERS AND FABRYS-DISEASE

1 January 1977

journal article
research article

Vol. 7 (3) , 251-253

Abstract

Glucocerebrosidase and ceramidetrihexoside-.alpha.-galactosidase were obtained in a high degree of purity from humans placental tissue. The enzymes were infused in Gaucher and Fabry patients, respectively. Following the administration of the proteins to supplement the genetically determined deficiencies, there resulted a specific reduction in the accumulated glycolipids in the circulation and liver. Enzyme replacement may provide hope for the clinical treatment of these disorders.

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