Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease. (Q42638927)

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4 January 2020

title

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

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4 January 2020

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3

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Robin Lachmann

7

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Martin Hrebicek

8

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Frances Platt

9

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author name string

D Elstein

1

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C Hollak

2

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S van Weely

4

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M Maas

5

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T M Cox

6

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T D Butters

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R A Dwek

11

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A Zimran

12

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publication date

1 January 2004

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Journal of Inherited Metabolic Disease

4 January 2020

published in

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volume

27

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page(s)

757-766

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issue

6

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https://scigraph.springernature.com/pub.10.1023/b:boli.0000045756.54006.17

4 January 2020

exact match

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12 December 2020

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

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

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Effect of low-dose enzyme replacement therapy on bones in Gaucher disease patients with severe skeletal involvement

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Substrate reduction therapy in mouse models of the glycosphingolipidoses

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12 December 2020

Clinically relevant therapeutic endpoints in type I Gaucher disease.

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12 December 2020

Quantification of skeletal involvement in adults with type I Gaucher's disease: fat fraction measured by Dixon quantitative chemical shift imaging as a valid parameter

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12 December 2020

Management of neuronopathic Gaucher disease: a European consensus.

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12 December 2020

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

1 reference

12 December 2020