Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's Disease

19911,266 citationsJournal Articlebronze Open Access

Authors

Norman W. Barton · National Institute of Neurological Disorders and Stroke

Roscoe O. Brady

James M. Dambrosia

Adrian M. Di Bisceglie

Abstract

Intravenous administration of macrophage-targeted glucocerebrosidase produces objective clinical improvement in patients with type 1 Gaucher's disease. The hematologic and visceral responses to enzyme replacement develop more rapidly than the skeletal response.

Topics & Keywords

Lysosomal Storage Disorders Research Carbohydrate Chemistry and Synthesis Cellular transport and secretion

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: New England Journal of Medicine

Volume 324, Issue 21, pp. 1464-1470

DOI: 10.1056/nejm199105233242104

Field-Weighted Citation Impact: 18.30