(Q46933420)

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

In more languages

Statements

scholarly article

1 reference

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)

1 reference

Europe PubMed Central

26 December 2017

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

alglucosidase alfa

1 reference

based on heuristic

inferred from title

Benedikt Schoser

5

1 reference

Europe PubMed Central

26 December 2017

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

14

object named as

Volker Straub

1 reference

Europe PubMed Central

26 December 2017

Beth L. Thurberg

22

object named as

Beth L Thurberg

1 reference

Europe PubMed Central

26 December 2017

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

author name string

Ans van der Ploeg

1

1 reference

Europe PubMed Central

26 December 2017

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

Pierre G Carlier

2

1 reference

Europe PubMed Central

26 December 2017

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

Robert-Yves Carlier

3

1 reference

Europe PubMed Central

26 December 2017

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

John T Kissel

4

1 reference

Europe PubMed Central

26 December 2017

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

Stephan Wenninger

6

1 reference

Europe PubMed Central

26 December 2017

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

Alan Pestronk

7

1 reference

Europe PubMed Central

26 December 2017

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

Richard J Barohn

8

1 reference

Europe PubMed Central

26 December 2017

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

Mazen M Dimachkie

9

1 reference

Europe PubMed Central

26 December 2017

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

Ozlem Goker-Alpan

10

1 reference

Europe PubMed Central

26 December 2017

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

Tahseen Mozaffar

11

1 reference

Europe PubMed Central

26 December 2017

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

Loren D M Pena

12

1 reference

Europe PubMed Central

26 December 2017

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

Zachary Simmons

13

1 reference

Europe PubMed Central

26 December 2017

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

Michela Guglieri

15

1 reference

Europe PubMed Central

26 December 2017

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

Peter Young

16

1 reference

Europe PubMed Central

26 December 2017

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

Matthias Boentert

17

1 reference

Europe PubMed Central

26 December 2017

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

Pierre-Yves Baudin

18

1 reference

Europe PubMed Central

26 December 2017

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

Stephan Wens

19

1 reference

Europe PubMed Central

26 December 2017

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

Raheel Shafi

20

1 reference

Europe PubMed Central

26 December 2017

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

Carl Bjartmar

21

1 reference

Europe PubMed Central

26 December 2017

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

publication date

19 May 2016

1 reference

Europe PubMed Central

26 December 2017

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

Molecular Genetics and Metabolism

1 reference

Europe PubMed Central

26 December 2017

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

119

1 reference

Europe PubMed Central

26 December 2017

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

1-2

1 reference

Europe PubMed Central

26 December 2017

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

115-123

1 reference

Europe PubMed Central

26 December 2017

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

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

Sitelinks

Wikipedia(0 entries)

Wikibooks(0 entries)

Wikinews(0 entries)

Wikiquote(0 entries)

Wikisource(0 entries)

Wikiversity(0 entries)

Wikivoyage(0 entries)

Wiktionary(0 entries)

Multilingual sites(0 entries)

Retrieved from "https://www.wikidata.org/w/index.php?title=Q46933420&oldid=2138501988"