European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience. (Q39287288)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28477382%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

review article

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title

European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28477382%20AND%20SRC:MED&resulttype=core&format=json

enzyme replacement therapy

19 March 2020

main subject

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author

Ans van der Ploeg

1

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19 March 2020

Robin Lachmann

6

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19 March 2020

Samuel Ignacio Pascual

7

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19 March 2020

Thomas M Stulnig

10

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19 March 2020

author name string

M E Kruijshaar

2

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19 March 2020

A Toscano

3

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19 March 2020

P Laforêt

4

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19 March 2020

C Angelini

5

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19 March 2020

M Roberts

8

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19 March 2020

K Rösler

9

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19 March 2020

P A van Doorn

11

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19 March 2020

P Y K Van den Bergh

12

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19 March 2020

J Vissing

13

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19 March 2020

B Schoser

14

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19 March 2020

European Pompe Consortium

15

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19 March 2020

publication date

6 May 2017

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European Journal of Neurology

19 March 2020

published in

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19 March 2020

volume

24

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19 March 2020

issue

6

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19 March 2020

page(s)

768-e31

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

19 March 2020

cites work

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Consensus treatment recommendations for late-onset Pompe disease

21 January 2018

1 reference

Diagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert group

21 January 2018

1 reference

Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease.

21 January 2018

1 reference

Diagnosis and therapy of late onset Pompe disease

21 January 2018

1 reference

208th ENMC International Workshop: Formation of a European Network to develop a European data sharing model and treatment guidelines for Pompe disease Naarden, The Netherlands, 26-28 September 2014

21 January 2018

1 reference

Enzyme replacement therapy and fatigue in adults with Pompe disease

21 January 2018

1 reference

Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up

21 January 2018

1 reference

Enzyme replacement therapy improves respiratory outcomes in patients with late-onset type II glycogenosis and high ventilator dependency.

21 January 2018

1 reference

Ten years of the international Pompe survey: patient reported outcomes as a reliable tool for studying treated and untreated children and adults with non-classic Pompe disease

21 January 2018

1 reference

Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years.

21 January 2018

1 reference

Prevalence of Pompe disease in 3,076 patients with hyperCKemia and limb-girdle muscular weakness.

21 January 2018

1 reference

From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides

21 January 2018

1 reference

Progress in Enzyme Replacement Therapy in Glycogen Storage Disease Type II

21 January 2018

1 reference

Should patients with asymptomatic pompe disease be treated? A nationwide study in France

21 January 2018

1 reference

Making diagnosis of Pompe disease at a presymptomatic stage: to treat or not to treat?

21 January 2018

1 reference

Prognostic factors for the late onset Pompe disease with enzyme replacement therapy: from our experience of 4 cases including an autopsy case.

21 January 2018

1 reference

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

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

21 January 2018

1 reference

Use of the muscle volume analyzer to evaluate enzyme replacement therapy in late-onset Pompe disease.

21 January 2018

1 reference

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

21 January 2018

1 reference

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

21 January 2018

1 reference

Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease

21 January 2018

1 reference

Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy.

21 January 2018

1 reference

The impact of antibodies in late-onset Pompe disease: a case series and literature review

21 January 2018

1 reference

Pompe disease: literature review and case series

21 January 2018

1 reference

Pregnancy during enzyme replacement therapy for late-onset acid maltase deficiency.

21 January 2018

1 reference

Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Fibromyalgia-like symptoms associated with irritable bowel syndrome: A challenging diagnosis of late-onset Pompe disease.

21 January 2018

1 reference

New motor outcome function measures in evaluation of late-onset Pompe disease before and after enzyme replacement therapy

21 January 2018

1 reference

Enzyme replacement therapy in late-onset type II glycogenosis.

21 January 2018

1 reference

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

Exercise testing in late-onset glycogen storage disease type II patients undergoing enzyme replacement therapy.

21 January 2018

1 reference

Clinical and molecular aspects of 30 patients with late-onset Pompe disease (LOPD): unusual features and response to treatment

21 January 2018

1 reference

Role of the cardio-pulmonary exercise test and six-minute walking test in the evaluation of exercise performance in patients with late-onset Pompe disease.

21 January 2018

1 reference

Sixty six-month follow-up of muscle power and respiratory function in a case with adult-type Pompe disease treated with enzyme replacement therapy

21 January 2018

1 reference

Effect of enzyme replacement therapy in late onset Pompe disease: open pilot study of 48 weeks follow-up.

21 January 2018

1 reference

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

21 January 2018

1 reference

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

Effect of enzyme replacement therapy on isokinetic strength for all major muscle groups in four patients with Pompe disease—a long-term follow-up

21 January 2018

1 reference

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

21 January 2018

Identifiers

DOI

10.1111/ENE.13285

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28477382%20AND%20SRC:MED&resulttype=core&format=json

release_grv7t6cl4beenbc4ad6eo3e4hi

19 March 2020

Fatcat ID

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Fatcat

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mapped directly with Wikidata item

24 November 2022

based on heuristic

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