Article Text

Download PDFPDF
Review
Miglustat as a therapeutic agent: prospects and caveats
  1. Rosemarie E Venier1,
  2. Suleiman A Igdoura1,2
  1. 1Department of Biology, McMaster University, Hamilton, Ontario, Canada
  2. 2Department of Pathology and Molecular Medicine, McMaster University, Hamilton, Ontario, Canada
  1. Correspondence to Professor Suleiman A Igdoura, McMaster University, 1280 Main St. W. LSB 335, Hamilton, Ontario L8S 4K1, Canada; igdoura{at}mcmaster.ca

Abstract

A viable treatment for lysosomal storage disease has been very difficult to attain. One option is pharmacological inhibition of synthetic pathways to reduce substrate accumulations. Miglustat N-butyldeoxynojirimycin (NBDNJ), an inhibitor of glucosylceramide synthase, has shown much promise in clinical trials for the treatment of Type I Gaucher disease. The molecular events invoked by NBDNJ in cell culture and in animal models have not been so definitive. This review discusses the biochemical and molecular impact of NBDNJ as it relates to its potential as a therapeutic drug.

  • Metabolic disorders
  • Lipid disorders
  • Neurosciences
  • Movement disorders (other than Parkinsons)
  • Molecular genetics

Statistics from Altmetric.com

Request Permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.