Enzyme Replacement in Gaucher Disease
Open Access
- 30 November 2004
- journal article
- review article
- Published by Public Library of Science (PLoS) in PLoS Medicine
- Vol. 1 (2), e21
- https://doi.org/10.1371/journal.pmed.0010021
Abstract
The development of enzyme replacement therapy for Gaucher disease was a triumph of translational medicine. What were the key steps in its development? What are the controversies surrounding its use?Keywords
This publication has 25 references indexed in Scilit:
- Long-Term Safety and Efficacy of Enzyme Replacement Therapyfor Fabry DiseaseAmerican Journal of Human Genetics, 2004
- Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendationsThe Journal of Pediatrics, 2004
- A contradictory treatment for lysosomal storage disorders: inhibitors enhance mutant enzyme activityTrends in Pharmacological Sciences, 2003
- Commentary: Dosage–Response in the Treatment of Gaucher Disease by Enzyme Replacement TherapyBlood Cells, Molecules, and Diseases, 2000
- Effect of Low-dose Enzyme Replacement Therapy on Bones in Gaucher Disease Patients with Severe Skeletal InvolvementBlood Cells, Molecules, and Diseases, 1996
- Gaucher DiseaseMedicine, 1995
- The Clinical Course of Treated and Untreated Gaucher Disease. A Study of 45 PatientsBlood Cells, Molecules, and Diseases, 1995
- Gaucher disease: new molecular approaches to diagnosis and treatmentScience, 1992
- Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's DiseaseNew England Journal of Medicine, 1991
- Human β-glucuronidase: In vivo clearance and in vitro uptake by a glycoprotein recognition system on reticuloendothelial cellsCell, 1978