Treatment of a child diagnosed with Niemann-Pick disease type C with miglustat: a case report in Brazil (Q34860824)

4 August 2017

title

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

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Niemann-Pick disease, type C1

4 August 2017

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1 reference

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M L F Santos

1

1 reference

4 August 2017

S Raskin

2

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4 August 2017

D S Telles

3

1 reference

4 August 2017

A Löhr

4

1 reference

4 August 2017

P B N Liberalesso

5

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4 August 2017

S C Vieira

6

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4 August 2017

M L Cordeiro

7

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4 August 2017

publication date

21 October 2008

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Journal of Inherited Metabolic Disease

4 August 2017

published in

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4 August 2017

volume

31 Suppl 2

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4 August 2017

page(s)

S357-61

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The Child Behavior Checklist and related forms for assessing behavioral/emotional problems and competencies

4 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

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

https://api.crossref.org/works/10.1007/S10545-008-0923-9

Pharmacological and genetic modifications of somatic cholesterol do not substantially alter the course of CNS disease in Niemann-Pick C mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

The effect of cholesterol-lowering agents on hepatic and plasma cholesterol in Niemann-Pick disease type C.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

Niemann-Pick disease type C.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

Valproate-associated hyperammonemic encephalopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

Consequences of NPC1 and NPC2 loss of function in mammalian neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/S10545-008-0923-9

Inhibition of N-linked complex oligosaccharide formation by 1-deoxynojirimycin, an inhibitor of processing glucosidases

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/18937049

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

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

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/18937049

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

The adult form of Niemann-Pick disease type C.

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/18937049

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1007/S10545-008-0923-9

1 reference

Dimensions Publication ID

4 August 2017

1005240517

0 references

1 reference