(Q35710895)

English

Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study

scientific article published on 28 May 2015

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scholarly article

1 reference

Europe PubMed Central

9 August 2017

Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study (English)

1 reference

Europe PubMed Central

9 August 2017

Niemann-Pick disease

1 reference

based on heuristic

inferred from title

Niemann-Pick disease, type C1

1 reference

based on heuristic

inferred from title

Marc C. Patterson

object named as

Marc C Patterson

1

0 references

2

object named as

Eugen Mengel

1 reference

Europe PubMed Central

9 August 2017

author name string

Marie T Vanier

3

1 reference

Europe PubMed Central

9 August 2017

Barbara Schwierin

4

1 reference

Europe PubMed Central

9 August 2017

Audrey Muller

5

1 reference

Europe PubMed Central

9 August 2017

Peter Cornelisse

6

1 reference

Europe PubMed Central

9 August 2017

Mercè Pineda

7

1 reference

Europe PubMed Central

9 August 2017

NPC Registry investigators

8

1 reference

Europe PubMed Central

9 August 2017

language of work or name

0 references

publication date

28 May 2015

1 reference

Europe PubMed Central

9 August 2017

Orphanet Journal of Rare Diseases

1 reference

Europe PubMed Central

9 August 2017

10

1 reference

Europe PubMed Central

9 August 2017

1

0 references

65

1 reference

Europe PubMed Central

9 August 2017

copyright license

Creative Commons Attribution 4.0 International

28 May 2015

1 reference

April 2022 Public Data File from Crossref

copyright status

0 references

https://scigraph.springernature.com/pub.10.1186/s13023-015-0284-z

0 references

Disease and patient characteristics in NP-C patients: findings from an international disease registry

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

Use of miglustat in a child with late-infantile-onset Niemann-Pick disease type C and frequent seizures: a case report

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

Dysphagia as a risk factor for mortality in Niemann-Pick disease type C: systematic literature review and evidence from studies with miglustat

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

Recommendations for the diagnosis and management of Niemann-Pick disease type C: an update

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

Gastrointestinal disturbances and their management in miglustat-treated patients.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

Niemann-Pick disease type C

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

Niemann-Pick disease type C.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

12 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

25 September 2018

Dietary modifications in patients receiving miglustat.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

25 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

25 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

25 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

25 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

25 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4462071

25 September 2018

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/26017010

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1186/S13023-015-0284-Z

1 reference

Europe PubMed Central

9 August 2017

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0 references

1 reference

Europe PubMed Central

9 August 2017

1 reference

Europe PubMed Central

9 August 2017

ResearchGate publication ID

0 references

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