Enzyme replacement in Gaucher disease

PLoS Med. 2004 Nov;1(2):e21. doi: 10.1371/journal.pmed.0010021. Epub 2004 Nov 30.

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?

Publication types

  • Review

MeSH terms

  • Clinical Trials as Topic
  • Dose-Response Relationship, Drug
  • Gaucher Disease / complications
  • Gaucher Disease / drug therapy*
  • Gaucher Disease / physiopathology*
  • Glucosylceramidase / administration & dosage
  • Glucosylceramidase / therapeutic use*
  • Humans
  • Lectins, C-Type
  • Mannose Receptor
  • Mannose-Binding Lectins
  • Patient Selection
  • Receptors, Cell Surface
  • Treatment Outcome

Substances

  • Lectins, C-Type
  • Mannose Receptor
  • Mannose-Binding Lectins
  • Receptors, Cell Surface
  • Glucosylceramidase