(Q28547115)

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Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

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Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase (English)

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Daniel-G. Bichet

2

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Europe PubMed Central

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31 May 2020

5

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31 May 2020

David G Warnock

1

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David G Warnock

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31 May 2020

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Myrl Holida

3

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31 May 2020

Ozlem Goker-Alpan

4

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31 May 2020

Mark Thomas

6

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31 May 2020

Francois Eyskens

7

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31 May 2020

Suma Shankar

8

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31 May 2020

Mathews Adera

9

1 reference

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31 May 2020

Sheela Sitaraman

10

1 reference

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31 May 2020

Richie Khanna

11

1 reference

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31 May 2020

John J Flanagan

12

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31 May 2020

Brandon A Wustman

13

1 reference

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31 May 2020

Jay Barth

14

1 reference

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31 May 2020

Carrolee Barlow

15

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31 May 2020

Kenneth J Valenzano

16

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31 May 2020

David J Lockhart

17

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31 May 2020

Pol Boudes

18

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31 May 2020

Franklin K Johnson

19

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31 May 2020

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publication date

2015

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31 May 2020

10

1 reference

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31 May 2020

8

1 reference

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31 May 2020

e0134341

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31 May 2020

copyright license

Creative Commons Attribution 4.0 International

7 August 2015

1 reference

April 2022 Public Data File from Crossref

copyright status

0 references

A synthetic chaperone corrects the trafficking defect and disease phenotype in a protein misfolding disorder

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

19 March 2017

A phase 1/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

19 March 2017

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

29 September 2017

Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

29 September 2017

Enzyme therapy in Fabry disease: severe adverse events associated with anti-agalsidase cross-reactive IgG antibodies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

29 September 2017

The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

29 September 2017

The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in Fabry patient cell lines

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

29 September 2017

Agalsidase alfa and kidney dysfunction in Fabry disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

29 September 2017

Early therapeutic intervention in females with Fabry disease?

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

29 September 2017

Pharmacological chaperone corrects lysosomal storage in Fabry disease caused by trafficking-incompetent variants

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

29 September 2017

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

29 September 2017

Pharmacokinetics and Safety of Migalastat HCl and Effects on Agalsidase Activity in Healthy Volunteers.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

3 June 2018

Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

3 June 2018

A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

3 June 2018

Cellular and tissue localization of globotriaosylceramide in Fabry disease.

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0134341

21 January 2018

Natural history of Fabry renal disease: influence of alpha-galactosidase A activity and genetic mutations on clinical course

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0134341

21 January 2018

Enzyme replacement therapy improves peripheral nerve and sweat function in Fabry disease.

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0134341

21 January 2018

Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0134341

21 January 2018

Preclinical efficacy and safety of 1-deoxygalactonojirimycin in mice for Fabry disease

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0134341

21 January 2018

Relative bioavailability and the effect of meal type and timing on the pharmacokinetics of migalastat in healthy volunteers.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4529213

27 November 2018

In vitro inhibition and intracellular enhancement of lysosomal α-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives

1 reference

https://pubmed.ncbi.nlm.nih.gov/26252393

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Enzymatic Defect in Fabry's Disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/26252393

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Successful reinstitution of agalsidase beta therapy in Fabry disease patients with previous IgE-antibody or skin-test reactivity to the recombinant enzyme

1 reference

https://pubmed.ncbi.nlm.nih.gov/26252393

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Accelerated transport and maturation of lysosomal α–galactosidase A in Fabry lymphoblasts by an enzyme inhibitor

1 reference

https://pubmed.ncbi.nlm.nih.gov/26252393

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Cell-Based Screening of Active-Site Specific Chaperone for the Treatment of Fabry Disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/26252393

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1371/JOURNAL.PONE.0134341

2 references

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

Europe PubMed Central

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26252393%20AND%20SRC:MED&resulttype=core&format=json

31 May 2020

2015PLoSO..1034341W

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OpenCitations bibliographic resource ID

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

2 references

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

Europe PubMed Central

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26252393%20AND%20SRC:MED&resulttype=core&format=json

31 May 2020

2 references

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

Europe PubMed Central

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26252393%20AND%20SRC:MED&resulttype=core&format=json

31 May 2020

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