The role of the iminosugar <i>N</i>‐butyldeoxynojirimycin (miglustat) in the management of type I (non‐neuronopathic) Gaucher disease: A position statement

M. Cox, Timothy; M. F. G. Aerts, Johannes; Andria, Generoso; Beck, Michael; Belmatoug, Nadia; Bembi, Bruno; Chertkoff, Raul; vom Dahl, Stephan; Elstein, Deborah; Erikson, Anders; Giralt, M; Heitner, René; E. M. Hollak, Carla; Hrebicek, M.; E. Lewis, Susan; Mehta, Atul; M. Pastores, Gregory; Rolfs, A.; Sá-Miranda, Clara; Zimran, Ari

doi:10.1023/a:1025902113005

Public

The role of the iminosugar N‐butyldeoxynojirimycin (miglustat) in the management of type I (non‐neuronopathic) Gaucher disease: A position statement

Published in Journal of Inherited Metabolic Disease • Sep 1, 2003

Authors:

Timothy M. Cox

Johannes M. F. G. Aerts

Generoso Andria

Abstract

N-Butyldeoxynojirimycin (NB-DNJ, miglustat 'Zavesca') is an orally active iminosugar which inhibits the biosynthesis of macromolecular substrates that accumulate pathologically in glycosphingolipidoses. Clinical trials of NB-DNJ in patients with Gaucher's disease demonstrate the therapeutic potentia...

Subject

Substrate reduction therapy

Iminosugar

Glucocerebrosidase

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