Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease (Q30489156)

19 August 2017

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

1 reference

19 August 2017

9

Beth L Thurberg

1 reference

19 August 2017

Priya Sunil Kishnani

1

1 reference

19 August 2017

Marc Nicolino

2

1 reference

19 August 2017

Thomas Voit

3

1 reference

19 August 2017

R Curtis Rogers

4

1 reference

19 August 2017

Anne Chun-Hui Tsai

5

1 reference

19 August 2017

John Waterson

6

1 reference

19 August 2017

Gail E Herman

7

1 reference

19 August 2017

Andreas Amalfitano

8

1 reference

19 August 2017

Susan Richards

10

1 reference

19 August 2017

Mark Davison

11

1 reference

19 August 2017

Deyanira Corzo

12

1 reference

19 August 2017

Y T Chen

13

1 reference

19 August 2017

language of work or name

English

0 references

publication date

1 July 2006

1 reference

The Journal of Pediatrics

19 August 2017

1 reference

19 August 2017

149

1 reference

19 August 2017

1

1 reference

19 August 2017

89-97

1 reference

Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice

19 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

High-resolution light microscopy (HRLM) and digital analysis of Pompe disease pathology

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Direct multiplex assay of lysosomal enzymes in dried blood spots for newborn screening

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Long-term safety and efficacy of enzyme replacement therapy for Fabry disease

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Pompe disease in infants and children

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Towards a molecular therapy for glycogen storage disease type II (Pompe disease).

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counselling

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Left ventricular myocardial mass determined by cross-sectional echocardiography in normal newborns, infants, and children

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Quantitation of Mr 46000 and Mr 300000 mannose 6-phosphate receptors in human cells and tissues

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Expression of the insulin-like growth factor-II/mannose-6-phosphate receptor in multiple human tissues during fetal life and early infancy

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Glycogen storage disease type II: enzymatic screening in dried blood spots on filter paper

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Enzyme replacement therapy in the mouse model of Pompe disease

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2692727

Carrier frequency for glycogen storage disease type II in New York and estimates of affected individuals born with the disease

26 November 2018

1 reference

12 December 2020

Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: induction of humoral tolerance in seroconverted patients after repeat administration

1 reference

12 December 2020

Molecular genetic study of Pompe disease in Chinese patients in Taiwan

1 reference

12 December 2020

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

1 reference

12 December 2020

Human acid alpha-glucosidase from rabbit milk has therapeutic effect in mice with glycogen storage disease type II

1 reference

12 December 2020

10.1016/J.JPEDS.2006.02.035

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30 July 2022

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30 July 2022

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