Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2003;26(6):513-26.
doi: 10.1023/a:1025902113005.

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

T M Cox  1 , J M F G Aerts, G Andria, M Beck, N Belmatoug, B Bembi, R Chertkoff, S Vom Dahl, D Elstein, A Erikson, M Giralt, R Heitner, C Hollak, M Hrebicek, S Lewis, A Mehta, G M Pastores, A Rolfs, M C Sa Miranda, A Zimran; Advisory Council to the European Working Group on Gaucher Disease
Affiliations
Review

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

T M Cox et al. J Inherit Metab Dis. 2003.

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 potential of such substrate inhibitors in the glycolipid storage disorders. However, macrophage-targetted enzyme replacement using intravenous mannose-terminated 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-neuronopathic) Gaucher disease is evaluated with particular reference to the emerging role of oral N-butyldeoxynojirimycin (miglustat) as a substrate-reducing agent. This position statement represents the consensus viewpoint of an independent international advisory council to the European Working Group on Gaucher Disease.

PubMed Disclaimer

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Baillieres Clin Haematol. 1997 Dec;10(4):657-89 - PubMed
    1. Qual Life Res. 1998 Jul;7(5):373-86 - PubMed
    1. Expert Opin Investig Drugs. 2001 Mar;10(3):455-66 - PubMed
    1. Lancet. 2000 Apr 29;355(9214):1481-5 - PubMed
    1. J Inherit Metab Dis. 2000 Feb;23(1):77-82 - PubMed