(Q37012183)

17 August 2017

Atypical immunologic response in a patient with CRIM-negative Pompe disease (English)

1 reference

17 August 2017

1 reference

Mary-Alice Abbott

1

1 reference

17 August 2017

Sean N Prater

2

1 reference

17 August 2017

Suhrad G Banugaria

3

1 reference

17 August 2017

Susan M Richards

4

1 reference

17 August 2017

Sarah P Young

5

1 reference

17 August 2017

Amy S Rosenberg

6

1 reference

17 August 2017

Priya S Kishnani

7

1 reference

17 August 2017

11 August 2011

1 reference

Molecular Genetics and Metabolism

17 August 2017

1 reference

17 August 2017

104

1 reference

17 August 2017

4

1 reference

17 August 2017

583-586

1 reference

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

17 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

Arrhythmias in patients receiving enzyme replacement therapy for infantile Pompe disease

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

Immune response to enzyme replacement therapy in lysosomal storage disorder patients and animal models.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

The conservative substitution Asp-645-->Glu in lysosomal alpha-glucosidase affects transport and phosphorylation of the enzyme in an adult patient with glycogen-storage disease type II.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

CRIM-negative infantile Pompe disease: 42-month treatment outcome.

27 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

Long-term monitoring of patients with infantile-onset Pompe disease on enzyme replacement therapy using a urinary glucose tetrasaccharide biomarker.

4 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

4 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

Elimination of antibodies to recombinant enzyme in Pompe's disease.

4 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

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

4 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3711241

10.1016/J.YMGME.2011.08.003

4 September 2018

Identifiers

DOI

1 reference

17 August 2017

1 reference

17 August 2017

1 reference