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

@article{Cox2004TheRO,
  title={The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: A position statement},
  author={Timothy M. Cox and Johannes Aerts and Generoso Andria and Michael Beck and Nadia Belmatoug and Bruno Bembi and Raul Chertkoff and Stephan vom Dahl and Deborah Elstein and Anders Erikson and Manuel Giralt and Rene Heitner and Carla E. M. Hollak and Martin Hřeb{\'i}{\vc}ek and S. Lewis and Atual Mehta and Gregory M. Pastores and Arndt Rolfs and Maria Clara S{\'a} Miranda and Ari Zimran},
  journal={Journal of Inherited Metabolic Disease},
  year={2004},
  volume={26},
  pages={513-526}
}
N-Butyldeoxynojirimycin (NB-DNJ, miglustat 'Zavesca') is an orallyactive 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… 

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Substrate reduction therapy of glycosphingolipid storage disorders

Oral administration of an inhibitor of glucosylceramide synthesis (N-butyldeoxynojirimycin, registered in Europe since 2002 as miglustat (Zavesca), is effective in reversing clinical symptoms in type I Gaucher patients with mild to moderate disease manifestations.

Review of miglustat for clinical management in Gaucher disease type 1

  • C. Ficicioglu
  • Medicine, Biology
    Therapeutics and clinical risk management
  • 2008
The results of clinical studies and use of miglustat as a therapeutic agent in patients with type I Gaucher disease are discussed.
...

References

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Oral administration of an inhibitor of glucosylceramide synthesis (N-butyldeoxynojirimycin, registered in Europe since 2002 as miglustat (Zavesca), is effective in reversing clinical symptoms in type I Gaucher patients with mild to moderate disease manifestations.

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