Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C. (Q43607199)

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28 January 2020

title

Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C (English)

1 reference

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28 January 2020

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3

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Wuh-liang Hwu

9

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28 January 2020

Yin-Hsiu Chien

1

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28 January 2020

Ni-Chung Lee

4

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28 January 2020

Li-Kai Tsai

5

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author name string

S F Peng

2

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A C Huang

6

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S C Su

7

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C C Tseng

8

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28 January 2020

publication date

5 April 2012

1 reference

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Journal of Inherited Metabolic Disease

28 January 2020

published in

1 reference

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28 January 2020

volume

36

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issue

1

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page(s)

129-137

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https://scigraph.springernature.com/pub.10.1007/s10545-012-9479-9

28 January 2020

exact match

0 references

cites work

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

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.

21 January 2018

1 reference

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Intensive immunosuppression in progressive multiple sclerosis. A randomized, three-arm study of high-dose intravenous cyclophosphamide, plasma exchange, and ACTH.

21 January 2018

1 reference

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Isolated splenomegaly as the presenting feature of Niemann-Pick disease type C

21 January 2018

1 reference

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Niemann-Pick disease type C in adults

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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Niemann-Pick disease type C: diagnosis and outcome in children, with particular reference to liver disease.

21 January 2018

1 reference

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

21 January 2018

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The value of the Mini-Mental State Examination in childhood: a preliminary study.

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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Clinical experience with miglustat therapy in pediatric patients with Niemann-Pick disease type C: a case series

21 January 2018

1 reference

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A penetration-aspiration scale

21 January 2018

1 reference

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The adult form of Niemann-Pick disease type C.

21 January 2018

1 reference

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Complementation studies in Niemann-Pick disease type C indicate the existence of a second group

21 January 2018

1 reference

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Niemann-Pick Type C disease: characterizing lipid levels in patients with variant lysosomal cholesterol storage

21 January 2018

1 reference

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Niemann-Pick disease type C

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9479-9

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9479-9

The videofluoroscopic swallowing study shows a sustained improvement of dysphagia in children with Niemann-Pick disease type C after therapy with miglustat

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22476655

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Assessment of a modified Mini-Mental Scale for cognitive functions in children.

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22476655

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Quantifying swallowing function after stroke: A functional dysphagia scale based on videofluoroscopic studies

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22476655

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1007/S10545-012-9479-9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22476655%20AND%20SRC:MED&resulttype=core&format=json

Dimensions Publication ID

28 January 2020

0 references

1 reference

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