Enzyme Replacement Therapy for Gaucher Disease: Skeletal Responses to Macrophage-targeted Glucocerebrosidase

1995191 citationsJournal Article

Authors

Daniel I. Rosenthal · Massachusetts General Hospital

Samuel H. Doppelt

Abstract

Prolonged treatment over 3 1/2 years with macrophage-targeted glucocerebrosidase produces objective reversal of disease in both the axial and appendicular skeleton in patients with Gaucher disease. Marked improvement occurs in marrow composition and bone mass in both children and adults.

Topics & Keywords

Lysosomal Storage Disorders Research Glycosylation and Glycoproteins Research Cellular transport and secretion

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: PEDIATRICS

Volume 96, Issue 4, pp. 629-637

DOI: 10.1542/peds.96.4.629

Field-Weighted Citation Impact: 4.61