Immune Tolerance Strategies in Siblings with Infantile Pompe Disease-Advantages for a Preemptive Approach to High-Sustained Antibody Titers (Q35836295)

10 August 2017

Immune Tolerance Strategies in Siblings with Infantile Pompe Disease-Advantages for a Preemptive Approach to High-Sustained Antibody Titers (English)

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10 August 2017

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2

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14 June 2020

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Priya Kishnani

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14 June 2020

author name string

Elizabeth O Stenger

series ordinal

1

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26167453%20AND%20SRC:MED&resulttype=core&format=json

14 June 2020

Emily Lisi

series ordinal

3

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14 June 2020

Michael J Gambello

series ordinal

4

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14 June 2020

publication date

1 September 2015

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Molecular Genetics and Metabolism Reports

10 August 2017

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26167453%20AND%20SRC:MED&resulttype=core&format=json

14 June 2020

volume

4

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14 June 2020

page(s)

30-34

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Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease

14 June 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

The emerging phenotype of long-term survivors with infantile Pompe disease.

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease.

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

Therapeutic potential of proteasome inhibition in Duchenne and Becker muscular dystrophies

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

Pompe disease diagnosis and management guideline.

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

A phase 2 study of bortezomib in relapsed, refractory myeloma

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

The proteasome inhibitor bortezomib depletes plasma cells and protects mice with lupus-like disease from nephritis

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

Reduction of skeletal muscle atrophy by a proteasome inhibitor in a rat model of denervation

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

Sibling phenotype concordance in classical infantile Pompe disease

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4497810

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

22 October 2018

1 reference

Crossref

https://api.crossref.org/works/10.1016%2FJ.YMGMR.2015.05.004

Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease

7 January 2021

1 reference

Crossref

https://api.crossref.org/works/10.1016%2FJ.YMGMR.2015.05.004

10.1016/J.YMGMR.2015.05.004

7 January 2021

Identifiers

DOI

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26167453%20AND%20SRC:MED&resulttype=core&format=json

14 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26167453%20AND%20SRC:MED&resulttype=core&format=json

14 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26167453%20AND%20SRC:MED&resulttype=core&format=json