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Clinical trials of NB\u2010DNJ in patients with Gaucher's disease demonstrate the therapeutic potential of such substrate inhibitors in the glycolipid storage disorders. However, macrophage\u2010targetted enzyme replacement using intravenous mannose\u2010terminated human glucocerebrosidase (imiglucerase, Cerezyme) is highly effective in ameliorating many of the manifestations of Gaucher's disease and is a treatment in widespread use. Given that imiglucerase and miglustat are now both licensed for the treatment of Gaucher's disease, there is a need to review their therapeutic status. Here the treatment of type 1 (non\u2010neuronopathic) Gaucher disease is evaluated with particular reference to the emerging role of oral <jats:italic>N<\/jats:italic>\u2010butyldeoxynojirimycin (miglustat) as a substrate\u2010reducing agent. This position statement represents the consensus viewpoint of an independent international advisory council to the European Working Group on Gaucher Disease.<\/jats:p>","DOI":"10.1023\/a:1025902113005","type":"journal-article","created":{"date-parts":[[2003,10,24]],"date-time":"2003-10-24T22:52:54Z","timestamp":1067035974000},"page":"513-526","source":"Crossref","is-referenced-by-count":206,"title":["The role of the iminosugar <i>N<\/i>\u2010butyldeoxynojirimycin (miglustat) in the management of type I (non\u2010neuronopathic) Gaucher disease: A position statement"],"prefix":"10.1002","volume":"26","author":[{"given":"T. M.","family":"Cox","sequence":"first","affiliation":[{"name":"Department of Medicine University of Cambridge, Addenbrooke's Hospital  Cambridge UK"},{"name":"Department of Medicine University of Cambridge  Box 157, Addenbrooke's Hospital Cambridge CB2 2QQ UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J. M. F. 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