Therapy for Gaucher disease: don't stop thinking about tomorrow

Mol Genet Metab. 2007 Feb;90(2):122-5. doi: 10.1016/j.ymgme.2006.09.007. Epub 2006 Nov 3.


While enzyme replacement therapy for Gaucher disease has been widely used and appears to be an efficacious and safe treatment, this success should not be a reason for complacency. Other treatment strategies currently under consideration for patients with Gaucher disease include gene therapy, substrate reduction therapy and chaperone therapy. Furthermore, improvements in enzyme therapy could also have a significant clinical impact. Individuals with Gaucher disease and other lysosomal disorders will greatly benefit from continual refinement and optimization of the current therapy, as well as from the development of new treatment modalities that offer improvements in efficacy, cost, safety and availability.

Publication types

  • Research Support, N.I.H., Intramural
  • Review
  • Comment

MeSH terms

  • Gaucher Disease / drug therapy
  • Gaucher Disease / therapy*
  • Genetic Therapy / methods
  • Glucosylceramidase / therapeutic use
  • Glycosphingolipids / antagonists & inhibitors
  • Humans
  • Stem Cell Transplantation


  • Glycosphingolipids
  • Glucosylceramidase
  • imiglucerase