(Q34641771)

3 August 2017

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

1 reference

enzyme replacement therapy

3 August 2017

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Benedikt Schoser

2

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ORCID Public Data File 2021

author name string

Antonio Toscano

series ordinal

1

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3 August 2017

28 August 2012

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3 August 2017

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3 August 2017

260

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3 August 2017

951-959

1 reference

3 August 2017

4

1 reference

https://scigraph.springernature.com/pub.10.1007/s00415-012-6636-x

3 August 2017

0 references

cites work

Progress in Enzyme Replacement Therapy in Glycogen Storage Disease Type II

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counselling

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Early detection of Pompe disease by newborn screening is feasible: results from the Taiwan screening program.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Consensus treatment recommendations for late-onset Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Modulation of glycogen synthesis by RNA interference: towards a new therapeutic approach for glycogenosis type II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Disease severity in children and adults with Pompe disease related to age and disease duration.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

The clinical and electrodiagnostic characteristics of Pompe disease with post-enzyme replacement therapy findings

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

A novel mutation of the GAA gene in a Finnish late-onset Pompe disease patient: clinical phenotype and follow-up with enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Glycogen storage disease type II (Pompe disease)--influence of enzyme replacement therapy in adults

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Pompe disease: from new views on pathophysiology to innovative therapeutic strategies

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Changes in nutritional status and body composition during enzyme replacement therapy in adult-onset type II glycogenosis

21 January 2018

1 reference

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Changes in skeletal muscle qualities during enzyme replacement therapy in late-onset type II glycogenosis: temporal and spatial pattern of mass vs. strength response.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

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

21 January 2018

1 reference

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Long-term enzyme replacement therapy for pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells.

21 January 2018

1 reference

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Toward deconstructing the phenotype of late-onset Pompe disease.

21 January 2018

1 reference

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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

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

21 January 2018

1 reference

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Effect of enzyme therapy in juvenile patients with Pompe disease: a three-year open-label study

21 January 2018

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

21 January 2018

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

21 January 2018

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24-months results in two adults with Pompe disease on enzyme replacement therapy.

21 January 2018

1 reference

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Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up

21 January 2018

1 reference

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The natural course of non-classic Pompe's disease; a review of 225 published cases

21 January 2018

1 reference

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Rapid progressive course of later-onset Pompe disease in Chinese patients.

21 January 2018

1 reference

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Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease

21 January 2018

1 reference

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Modification of the natural history of adult-onset acid maltase deficiency by nutrition and exercise therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Effects of enzyme replacement therapy on five patients with advanced late-onset glycogen storage disease type II: a 2-year follow-up study

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00415-012-6636-X

Carrier frequency for glycogen storage disease type II in New York and estimates of affected individuals born with the disease

21 January 2018

1 reference

12 December 2020

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

1 reference

12 December 2020

First experience with enzyme replacement therapy during pregnancy and lactation in Pompe disease

1 reference

12 December 2020

Effect of aerobic and resistance exercise training on late-onset Pompe disease patients receiving enzyme replacement therapy

1 reference

12 December 2020

Adult Pompe disease: clinical manifestations and outcome of the first Greek patients receiving enzyme replacement therapy

1 reference

12 December 2020

Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease

1 reference

12 December 2020

Increased inspiratory and expiratory muscle strength following respiratory muscle strength training (RMST) in two patients with late-onset Pompe disease

1 reference

12 December 2020

10.1007/S00415-012-6636-X

Identifiers

DOI

1 reference

PubMed ID

22926164

Dimensions Publication ID

3 August 2017

0 references

1 reference

PubMed ID

22926164