Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study (Q46933420)

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scientific article published on 19 May 2016

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English	Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study	scientific article published on 19 May 2016

Statements

scholarly article

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Europe PubMed Central

26 December 2017

http://europepmc.org/abstract/MED/27473031

Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study (English)

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Europe PubMed Central

26 December 2017

http://europepmc.org/abstract/MED/27473031

alglucosidase alfa

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based on heuristic

inferred from title

Benedikt Schoser

5

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object named as

Volker Straub

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Beth L. Thurberg

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object named as

Beth L Thurberg

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http://europepmc.org/abstract/MED/27473031

author name string

Ans van der Ploeg

1

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http://europepmc.org/abstract/MED/27473031

Pierre G Carlier

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http://europepmc.org/abstract/MED/27473031

Robert-Yves Carlier

3

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http://europepmc.org/abstract/MED/27473031

John T Kissel

4

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http://europepmc.org/abstract/MED/27473031

Stephan Wenninger

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http://europepmc.org/abstract/MED/27473031

Alan Pestronk

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Richard J Barohn

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Mazen M Dimachkie

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Ozlem Goker-Alpan

10

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Tahseen Mozaffar

11

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http://europepmc.org/abstract/MED/27473031

Loren D M Pena

12

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Zachary Simmons

13

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http://europepmc.org/abstract/MED/27473031

Michela Guglieri

15

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http://europepmc.org/abstract/MED/27473031

Peter Young

16

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http://europepmc.org/abstract/MED/27473031

Matthias Boentert

17

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http://europepmc.org/abstract/MED/27473031

Pierre-Yves Baudin

18

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http://europepmc.org/abstract/MED/27473031

Stephan Wens

19

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http://europepmc.org/abstract/MED/27473031

Raheel Shafi

20

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http://europepmc.org/abstract/MED/27473031

Carl Bjartmar

21

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http://europepmc.org/abstract/MED/27473031

publication date

19 May 2016

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Molecular Genetics and Metabolism

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115-123

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1-2

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alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe's disease).

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

The spectrum and diagnosis of acid maltase deficiency.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Pompe's disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

The stability of markers in dried-blood spots for recommended newborn screening disorders in the United States

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Pompe disease diagnosis and management guideline.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Consensus treatment recommendations for late-onset Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Late-onset Pompe disease primarily affects quality of life in physical health domains.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Juvenile and adult-onset acid maltase deficiency in France: genotype-phenotype correlation

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Cardiac evaluation in children and adults with Pompe disease sharing the common c.-32-13T>G genotype rarely reveals abnormalities

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Course of disability and respiratory function in untreated late-onset Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Validation of a generic approach to muscle water T2 determination at 3T in fat-infiltrated skeletal muscle

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Whole-body muscle MRI in 20 patients suffering from late onset Pompe disease: Involvement patterns

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Trunk muscle involvement in late-onset Pompe disease: Study of thirty patients

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Skeletal muscle quantitative nuclear magnetic resonance imaging follow-up of adult Pompe patients

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.013

7 January 2021

Identifiers

10.1016/J.YMGME.2016.05.013

1 reference

Europe PubMed Central

26 December 2017

http://europepmc.org/abstract/MED/27473031

1 reference

Europe PubMed Central

26 December 2017

http://europepmc.org/abstract/MED/27473031

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