Miglustat in adult and juvenile patients with Niemann-Pick disease type C: long-term data from a clinical trial (Q33444755)

26 July 2017

Miglustat in adult and juvenile patients with Niemann-Pick disease type C: long-term data from a clinical trial (English)

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Niemann-Pick disease, type C1

26 July 2017

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Larry Abel

4

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Marc C. Patterson

object named as

Marc C Patterson

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author name string

James E Wraith

1

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26 July 2017

Darleen Vecchio

2

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26 July 2017

Elizabeth Jacklin

3

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26 July 2017

Harbajan Chadha-Boreham

5

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26 July 2017

Cécile Luzy

6

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26 July 2017

Ruben Giorgino

7

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26 July 2017

31 December 2009

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Molecular Genetics and Metabolism

26 July 2017

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26 July 2017

99

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26 July 2017

4

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26 July 2017

351-357

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Prevalence of lysosomal storage disorders

26 July 2017

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Niemann-Pick disease type C.

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Lipid and cholesterol trafficking in NPC.

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Neurons in Niemann-Pick disease type C accumulate gangliosides as well as unesterified cholesterol and undergo dendritic and axonal alterations

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Niemann-Pick disease group C: clinical variability and diagnosis based on defective cholesterol esterification. A collaborative study on 70 patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Natural history of Niemann-Pick disease type C in a multicentre observational retrospective cohort study.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

The National Niemann-Pick C1 disease database: report of clinical features and health problems.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

The adult form of Niemann-Pick disease type C.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Long-term miglustat therapy in children with Niemann-Pick disease type C.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Miglustat in patients with Niemann-Pick disease Type C (NP-C): a multicenter observational retrospective cohort study

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

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

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Treatment of a child diagnosed with Niemann-Pick disease type C with miglustat: a case report in Brazil

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Linear clinical progression, independent of age of onset, in Niemann-Pick disease, type C

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Saccades in adult Niemann-Pick disease type C reflect frontal, brainstem, and biochemical deficits.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Recommendations on the diagnosis and management of Niemann-Pick disease type C.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2009.12.006

10.1016/J.YMGME.2009.12.006

7 January 2021

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DOI

1 reference

26 July 2017

1 reference