Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study (Q34661485)

3 August 2017

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

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Niemann–Pick disease, type C

3 August 2017

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Marc C Patterson

1

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3 August 2017

Darleen Vecchio

2

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3 August 2017

Helena Prady

3

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3 August 2017

Larry Abel

4

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3 August 2017

James E Wraith

5

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3 August 2017

1 September 2007

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3 August 2017

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3 August 2017

6

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3 August 2017

9

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3 August 2017

765-772

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Group C Niemann-Pick disease: faulty regulation of low-density lipoprotein uptake and cholesterol storage in cultured fibroblasts

3 August 2017

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Intracellular trafficking of Niemann-Pick C proteins 1 and 2: obligate components of subcellular lipid transport

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Niemann-Pick C1 disease: the I1061T substitution is a frequent mutant allele in patients of Western European descent and correlates with a classic juvenile phenotype

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Nine cases of sphingomyelin lipidosis, a new variant in Spanish-American Children. Juvenile variant of Niemann-Pick Disease with foamy and sea-blue histiocytes

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Niemann-Pick C1 disease gene: homology to mediators of cholesterol homeostasis

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Identification of HE1 as the second gene of Niemann-Pick C disease

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Cholesterol overload promotes morphogenesis of a Niemann-Pick C (NPC)-like compartment independent of inhibition of NPC1 or HE1/NPC2 function

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Secondary accumulation of gangliosides in lysosomal storage disorders

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

GM2 ganglioside and pyramidal neuron dendritogenesis

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

The Niemann-Pick C1 protein in recycling endosomes of presynaptic nerve terminals

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Consequences of NPC1 and NPC2 loss of function in mammalian neurons

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Niemann-Pick type C disease in two affected sisters: ocular motor recordings and brain-stem neuropathology

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

A riddle wrapped in a mystery: understanding Niemann-Pick disease, type C.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Bipolar Disorder and Niemann-Pick Disease Type C

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

The neuropsychiatry of Niemann-Pick type C disease in adulthood.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Niemann-Pick disease type C: cataplexy and hypocretin in cerebrospinal fluid

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Evidence for independent feedback control of horizontal and vertical saccades from Niemann-Pick type C disease.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Abnormal vertical optokinetic nystagmus in infants and children

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

A neurovisceral storage disease with vertical supranuclear ophthalmoplegia, and its relationship to Niemann-Pick disease. A report of nine patients.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Therapy of Niemann-Pick disease, type C.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

New developments in treating glycosphingolipid storage diseases.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Emerging strategies for the treatment of hereditary metabolic storage disorders

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Storage solutions: treating lysosomal disorders of the brain

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Eye movement abnormalities as a sign for the diagnosis in Niemann-Pick disease type C

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

On the identification and analysis of saccadic eye movements--a quantitative study of the processing procedures.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

Benzodiazepine pharmacodynamics: utility of eye movement measures.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

The natural history of Niemann-Pick disease type C in the UK.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2807%2970194-1

7 January 2021

10.1016/S1474-4422(07)70194-1

Identifiers

DOI

1 reference

3 August 2017

1 reference