36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy (Q42634296)

4 November 2017

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

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enzyme replacement therapy

4 November 2017

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12

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4 November 2017

11

Eugen Mengel

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4 November 2017

2

Cornelia Kornblum

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4 November 2017

Caroline Regnery

1

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4 November 2017

Frank Hanisch

3

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4 November 2017

Stefan Vielhaber

4

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4 November 2017

Nicola Strigl-Pill

5

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4 November 2017

Birgit Grunert

6

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4 November 2017

Wolfgang Müller-Felber

7

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4 November 2017

Franz Xaver Glocker

8

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4 November 2017

Matthias Spranger

9

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4 November 2017

Marcus Deschauer

10

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4 November 2017

31 January 2012

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Journal of Inherited Metabolic Disease

4 November 2017

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4 November 2017

35

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4 November 2017

837-845

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4 November 2017

5

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https://scigraph.springernature.com/pub.10.1007/s10545-012-9451-8

4 November 2017

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cites work

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

Neural deficits contribute to respiratory insufficiency in Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

Twenty-two novel mutations in the lysosomal ?-glucosidase gene (GAA) underscore the genotype-phenotype correlation in glycogen storage disease type II

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

Update of the Pompe disease mutation database with 107 sequence variants and a format for severity rating

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder--murine Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

The prevalence and impact of scoliosis in Pompe disease: lessons learned from the Pompe Registry

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

Therapeutic approaches in glycogen storage disease type II/Pompe Disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

Toward deconstructing the phenotype of late-onset Pompe disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

Late onset Pompe disease: Clinical and neurophysiological spectrum of 38 patients including long-term follow-up in 18 patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-012-9451-8

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

12 December 2020

Adult onset acid maltase deficiency. Distribution and progression of clinical and pathological abnormality in a family

1 reference

12 December 2020

High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa

1 reference

12 December 2020

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

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

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

1 reference

12 December 2020

Adult-onset glycogen storage disease type 2: clinico-pathological phenotype revisited

1 reference

12 December 2020

Rate of progression and predictive factors for pulmonary outcome in children and adults with Pompe disease

1 reference

12 December 2020

Molecular analysis and protein processing in late-onset Pompe disease patients with low levels of acid α-glucosidase activity

1 reference

12 December 2020

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

1 reference

12 December 2020

Role of autophagy in the pathogenesis of Pompe disease.

1 reference

12 December 2020

Molecular diagnosis of German patients with late-onset glycogen storage disease type II

1 reference

12 December 2020

Enzyme replacement therapy induces T-cell responses in late-onset Pompe disease

1 reference

12 December 2020

Improvement with ongoing Enzyme Replacement Therapy in advanced late-onset Pompe disease: A case study

1 reference

12 December 2020

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

1 reference

12 December 2020

10.1007/S10545-012-9451-8

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DOI

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Dimensions Publication ID

4 November 2017

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