24 month-treatment with miglustat of three patients with Niemann-Pick disease type C: follow up using brain spectroscopy (Q34600390)

3 August 2017

24 month-treatment with miglustat of three patients with Niemann-Pick disease type C: follow up using brain spectroscopy (English)

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Niemann-Pick disease, type C1

3 August 2017

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based on heuristic

inferred from title

author

Thierry Billette de Villemeur

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object named as

Thierry Billette de Villemeur

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3 August 2017

Damien Galanaud

1

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3 August 2017

Ayman Tourbah

2

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3 August 2017

Stéphane Lehéricy

3

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3 August 2017

Nathalie Leveque

4

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3 August 2017

Bénédicte Heron

5

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3 August 2017

Nathalie Guffon

7

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3 August 2017

François Feillet

8

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3 August 2017

Nicole Baumann

9

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3 August 2017

Marie T Vanier

10

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Frédéric Sedel

11

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3 August 2017

13 November 2008

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Molecular Genetics and Metabolism

3 August 2017

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3 August 2017

96

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3 August 2017

55-58

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3 August 2017

2

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Niemann-Pick disease type C.

3 August 2017

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Consequences of NPC1 and NPC2 loss of function in mammalian neurons

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.002

The adult form of Niemann-Pick disease type C.

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Storage solutions: treating lysosomal disorders of the brain

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Critical role for glycosphingolipids in Niemann-Pick disease type C.

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Treatment with miglustat reverses the lipid-trafficking defect in Niemann-Pick disease type C.

7 January 2021

1 reference

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Treatment of Niemann-Pick disease type C in two children with miglustat: initial responses and maintenance of effects over 1 year.

7 January 2021

1 reference

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Motion analysis of a child with Niemann-Pick disease type C treated with miglustat

7 January 2021

1 reference

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Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

7 January 2021

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

7 January 2021

1 reference

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Niemann-Pick C disease in Spain: clinical spectrum and development of a disability scale

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High field MR imaging and 1H-MR spectroscopy in clinically isolated syndromes suggestive of multiple sclerosis: correlation between metabolic alterations and diagnostic MR imaging criteria.

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Proton magnetic resonance spectroscopic imaging in the clinical evaluation of patients with Niemann-Pick type C disease

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Expanding the diagnostic and research toolbox for inborn errors of metabolism: the role of magnetic resonance spectroscopy

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Magnetic resonance spectroscopy changes following haemopoietic stem cell transplantation in children with cerebral adrenoleukodystrophy.

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10.1016/J.YMGME.2008.10.002

7 January 2021

Identifiers

DOI

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3 August 2017

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