Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. (Q53912470)

22 May 2018

title

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

1 reference

22 May 2018

1 reference

Martin Hrebícek

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Robin Lachmann

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Frances Platt

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Johannes Aerts

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Carla Hollak

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Deborah Elstein

object named as

Deborah Elstein

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Ari Zimran

object named as

Ari Zimran

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author name string

Timothy Cox

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Sonja van Weely

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Terry Butters

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Raymond Dwek

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Chris Moyses

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Irene Gow

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language of work or name

English

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publication date

1 April 2000

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22 May 2018

published in

The Lancet

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22 May 2018

volume

355

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22 May 2018

issue

9214

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22 May 2018

page(s)

1481-1485

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22 May 2018

Gaucher's disease: clinical features and natural history

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Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources

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Replacement therapy with imiglucerase for type 1 Gaucher's disease

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Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin

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Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin.

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Mice with disrupted GM2/GD2 synthase gene lack complex gangliosides but exhibit only subtle defects in their nervous system

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Plasma and metabolic abnormalities in Gaucher's disease.

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Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease

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7 January 2021

Low-dose low-frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type I Gaucher disease

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Elevated plasma chitotriosidase activity in various lysosomal storage disorders.

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Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

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N-butyldeoxygalactonojirimycin: a more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo

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7 January 2021

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Identifiers

DOI

1 reference

22 May 2018

1 reference

22 May 2018