Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis-Reference-Cited by-同舟云学术

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis

Published:2000-04 Issue:9214 Volume:355 Page:1481-1485
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

Cox Timothy,Lachmann Robin,Hollak Carla,Aerts Johannes,van Weely Sonja,Hrebícek Martin,Platt Frances,Butters Terry,Dwek Raymond,Moyses Chris,Gow Irene,Elstein Deborah,Zimran Ari

Publisher

Elsevier BV

Subject

General Medicine

Reference22 articles.

1. Glucocerebroside lipidoses: Gaucher disease;Beutler,1995

2. Gaucher disease: clinical, laboratory, radiologic, and genetic features of 53 patients;Zimran;Medicine (Baltimore),1992

3. Gaucher's disease: clinical features and natural history;Cox;Bailliere's Clin Haematol,1997

4. Enzyme therapy in nonneuronopathic Gaucher disease: comparative efficacy of mannoseterminated glucocerebrosidase from natural and recombinant sources;Grabowski;Ann Intern Med,1995

5. Replacement therapy with imiglucerase for non-neuronopathic Gaucher's disease;Zimran;Lancet,1995

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