(Q33442678)

26 July 2017

title

Pharmacokinetics, safety and tolerability of miglustat in the treatment of pediatric patients with GM2 gangliosidosis (English)

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26 July 2017

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1

Gustavo H B Maegawa

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26 July 2017

author name string

Paul L M van Giersbergen

2

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Sandra Yang

3

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Brenda Banwell

4

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Christopher P Morgan

5

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Jasper Dingemanse

6

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Cynthia J Tifft

7

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Joe T R Clarke

8

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26 July 2017

publication date

3 May 2009

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Molecular Genetics and Metabolism

26 July 2017

published in

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26 July 2017

volume

97

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26 July 2017

issue

4

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26 July 2017

page(s)

284-291

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Substrate reduction therapy for glycosphingolipid storage disorders

26 July 2017

cites work

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Rapid quantification of miglustat in human plasma and cerebrospinal fluid by liquid chromatography coupled with tandem mass spectrometry

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

7 January 2021

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The natural history of juvenile or subacute GM2 gangliosidosis: 21 new cases and literature review of 134 previously reported.

7 January 2021

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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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Allogeneic BMT followed by substrate reduction therapy in a child with subacute Tay-Sachs disease

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Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase

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

7 January 2021

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An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment

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7 January 2021

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Lysosomal unesterified cholesterol content correlates with liver cell death in murine Niemann-Pick type C disease

7 January 2021

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Cerebrospinal fluid biomarkers showing neurodegeneration in dogs with GM1 gangliosidosis: possible use for assessment of a therapeutic regimen

7 January 2021

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Diagnosis of Alzheimer's disease with cerebrospinal fluid tau protein and aspartate aminotransferase

7 January 2021

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7 January 2021

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Efficacy of cerebro-spinal fluid biochemistry in the diagnosis of brain insult

7 January 2021

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Gabapentin: a pooled analysis of adverse events from three clinical trials in patients with postherpetic neuralgia

7 January 2021

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Carbamazepine side effects in children and adults

7 January 2021

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7 January 2021

Identifiers

DOI

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26 July 2017

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