(Q42674306)

4 November 2017

title

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

1 reference

4 November 2017

Effect of enzyme therapy in juvenile patients with Pompe disease: A three-year open-label study (English)

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

7 July 2024

author

Nadine A van der Beek

2

object named as

N. A M E. van der Beek

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

author name string

C. I. van Capelle

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

M. L C. Hagemans

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

W. F M. Arts

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

W. C J. Hop

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

P. Lee

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

J. Jaeken

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

I. M E. Frohn-Mulder

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

P. J F M. Merkus

9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

D. Corzo

10

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

A. C. Puga

11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

A. J. Reuser

12

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

A. T. van der Ploeg

13

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?resulttype=core&format=json&query=SRC%3AMED%20AND%20EXT_ID%3A20817528

7 July 2024

language of work or name

English

1 reference

National Center for Biotechnology Information

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/esummary.fcgi?db=pubmed&retmode=json&id=20817528

7 July 2024

publication date

1 December 2010

1 reference

full work available at URL

4 November 2017

https://api.elsevier.com/content/article/PII:S0960896610005729?httpAccept=text/xml

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

https://api.elsevier.com/content/article/PII:S0960896610005729?httpAccept=text/plain

7 July 2024

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

7 July 2024

published in

Neuromuscular Disorders

1 reference

4 November 2017

volume

20

1 reference

4 November 2017

issue

12

1 reference

4 November 2017

page(s)

775-782

1 reference

glycogen storage disease II

4 November 2017

cites work

2

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

3

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease.

7 July 2024

4

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

5

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

6

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

7

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

8

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

9

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

10

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

11

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Identification of two subtypes of infantile acid maltase deficiency

7 July 2024

12

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Glycogenosis type II (acid maltase deficiency)

7 July 2024

13

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

14

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

15

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial

7 July 2024

16

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

17

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Enzyme replacement therapy in classical infantile pompe disease: results of a ten-month follow-up study

7 July 2024

18

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial

7 July 2024

19

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

20

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

21

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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.

7 July 2024

22

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Lung function testing: selection of reference values and interpretative strategies. American Thoracic Society

7 July 2024

23

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

24

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Reference values of maximum isometric muscle force obtained in 270 children aged 4–16 years by hand-held dynamometry

7 July 2024

27

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Hand-held myometry: reference values

7 July 2024

28

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Clinical trial in Duchenne dystrophy. I. The design of the protocol

7 July 2024

29

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

7 July 2024

ATS Statement

30

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

ATS/ERS Statement on respiratory muscle testing.

7 July 2024

32

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Spirometry in normal subjects in sitting, prone, and supine positions

7 July 2024

33

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Validation of two running tests as estimates of maximal aerobic power in children

7 July 2024

34

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Functional ability and muscle force in healthy children and ambulant Duchenne muscular dystrophy patients

7 July 2024

35

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Respiratory insufficiency and limb muscle weakness in adults with Pompe's disease

7 July 2024

36

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Respiratory failure in Pompe disease: treatment with noninvasive ventilation

7 July 2024

37

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Genotype-phenotype correlation in adult-onset acid maltase deficiency

7 July 2024

38

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Broad spectrum of Pompe disease in patients with the same c.-32-13T->G haplotype

7 July 2024

39

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Mutation profile of the GAA gene in 40 Italian patients with late onset glycogen storage disease type II

7 July 2024

40

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Development of measurement techniques

7 July 2024

41

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Relationship between two measures of upper extremity strength: manual muscle test compared to hand-held myometry

7 July 2024

43

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children.

7 July 2024

44

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

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

7 July 2024

45

1 reference

https://api.crossref.org/works/10.1016/J.NMD.2010.07.277

7 July 2024

Identifiers

1 reference

4 November 2017

1 reference