Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study. (Q51718537)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24906254%20AND%20SRC:MED&resulttype=core&format=json

20 March 2020

title

Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study (English)

1 reference

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enzyme replacement therapy

20 March 2020

main subject

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lysergic acid diethylamide

1 reference

6

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20 March 2020

Robin Lachmann

7

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20 March 2020

Stuart Logan

8

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20 March 2020

author name string

L J Anderson

1

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20 March 2020

W Henley

2

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20 March 2020

K M Wyatt

3

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20 March 2020

V Nikolaou

4

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20 March 2020

S Waldek

5

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20 March 2020

publication date

7 June 2014

1 reference

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Journal of Inherited Metabolic Disease

20 March 2020

published in

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20 March 2020

volume

37

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20 March 2020

issue

6

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20 March 2020

page(s)

945-952

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https://scigraph.springernature.com/pub.10.1007/s10545-014-9728-1

20 March 2020

exact match

0 references

cites work

Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

The spectrum and diagnosis of acid maltase deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Pompe disease diagnosis and management guideline.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Toward deconstructing the phenotype of late-onset Pompe disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Identification of two subtypes of infantile acid maltase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Low bone mass in Pompe disease: muscular strength as a predictor of bone mineral density

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

A randomized study of alglucosidase alfa in late-onset Pompe's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Genotype-phenotype correlation in adult-onset acid maltase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9728-1

Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II

21 January 2018

1 reference

12 December 2020

The NCS-LSD cohort study: a description of the methods and analyses used to assess the long-term effectiveness of enzyme replacement therapy and substrate reduction therapy in patients with lysosomal storage disorders.

1 reference

12 December 2020

Pompe disease: dramatic improvement in gastrointestinal function following enzyme replacement therapy. A report of three later-onset patients

1 reference

12 December 2020

Bone mineral density in adult patients with Pompe disease

1 reference

12 December 2020

10.1007/S10545-014-9728-1

Identifiers

DOI

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24906254%20AND%20SRC:MED&resulttype=core&format=json

release_ob35jwnxtje5fdmuwi2z6jns3a

20 March 2020

Fatcat ID

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Fatcat

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24 November 2022

mapped directly with Wikidata item

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