Therapy for Gaucher disease: don't stop thinking about tomorrow.
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.
Sidransky
Ellen
E
Section on Molecular Neurogenetics, Medical Genetics Branch, National Human Genome Research Institute, NIH, Building 35, Room 1A213, 35 Convent Drive, MSC 3708, Bethesda, MD 20892, USA. sidranse@mail.nih.gov
LaMarca
Mary E
ME
Ginns
Edward I
EI
D005776:Gaucher Disease / Q000188:drug therapy / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods; D005962:Glucosylceramidase / Q000627:therapeutic use; D006028:Glycosphingolipids / Q000037:antagonists & inhibitors; D006801:Humans; D033581:Stem Cell Transplantation
D006028:Glycosphingolipids; D005962:Glucosylceramidase; C090568:imiglucerase