Sustained therapeutic effects of oral miglustat (Zavesca,N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease-Reference-Cited by-同舟云学术

Sustained therapeutic effects of oral miglustat (Zavesca,N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease

Published:2004-11 Issue:6 Volume:27 Page:757-766
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Elstein D.¹,Hollak C.²,Aerts J. M. F. G.²,van Weely S.²,Maas M.²,Cox T. M.³,Lachmann R. H.³,Hrebicek M.⁴,Platt F. M.⁵,Butters T. D.⁵,Dwek R. A.⁵,Zimran A.¹

Affiliation:

1. ; Shaare Zedek Medical Centre; Jerusalem Israel

2. ; Academic Medical Centre; University of Amsterdam; Amsterdam The Netherlands

3. ; Department of Medicine; University of Cambridge, Addenbrooke's Hospital; Cambridge UK

4. Institute of Inherited Metabolic Disorders; Prague Czech Republic

5. ; Oxford Glycobiology Institute, Department of Biochemistry; University of Oxford; Oxford UK

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1023/B:BOLI.0000045756.54006.17/fullpdf

Reference14 articles.

1. Novel oral treatment of Gaucher ‘s disease with N-butyldeoxynojirimyci (OGT 918)to decrease substrate biosynthesis;Cox;Lancet,2000

2. The role of the iminosugar N-butyl-deoxy ojirimyci (miglustat)i the management of type 1 (non-neuronopathic)Gaucher disease:a positio statement;Cox;J Inherit Metab Dis,2003

3. Effect of low-dose ERT on bones in Gaucher disease patients with severe skeletal involvement;Elstein;Blood Cells Mol Dis,1996

4. Marked elevation of plasma chitotriosidase activity:a novel hallmark of Gaucher disease;Hollak;J lin Invest,1994

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