(Q34313390)

English

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

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scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

1 August 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 (English)

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

molecular chaperones

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1 reference

based on heuristic

inferred from title

1 reference

based on heuristic

inferred from title

pharmacodynamics

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based on heuristic

inferred from title

Roberto Giugliani

2

object named as

Roberto Giugliani

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Europe PubMed Central

PubMed publication ID

1 August 2017

author name string

Dominique P Germain

1

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Derralynn A Hughes

3

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Europe PubMed Central

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1 August 2017

Atul Mehta

4

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Europe PubMed Central

PubMed publication ID

1 August 2017

Kathy Nicholls

5

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Laura Barisoni

6

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Charles J Jennette

7

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Alexander Bragat

8

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Jeff Castelli

9

1 reference

Europe PubMed Central

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1 August 2017

Sheela Sitaraman

10

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

David J Lockhart

11

1 reference

Europe PubMed Central

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1 August 2017

Pol F Boudes

12

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Europe PubMed Central

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1 August 2017

publication date

24 November 2012

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Europe PubMed Central

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1 August 2017

Orphanet Journal of Rare Diseases

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Europe PubMed Central

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1 August 2017

7

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Europe PubMed Central

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1 August 2017

91

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Europe PubMed Central

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1 August 2017

https://scigraph.springernature.com/pub.10.1186/1750-1172-7-91

0 references

Novel quantitative method to evaluate globotriaosylceramide inclusions in renal peritubular capillaries by virtual microscopy in patients with fabry disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3527132

6 July 2018

A pharmacogenetic approach to identify mutant forms of α-galactosidase A that respond to a pharmacological chaperone for Fabry disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3527132

6 July 2018

Enzyme replacement therapy for Fabry disease: some answers but more questions

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3527132

6 July 2018

Progressive podocyte injury and globotriaosylceramide (GL-3) accumulation in young patients with Fabry disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3527132

6 July 2018

Pharmacological chaperone therapy by active-site-specific chaperones in Fabry disease: in vitro and preclinical studies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3527132

6 July 2018

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=3527132

6 July 2018

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=3527132

6 July 2018

Active-site-specific chaperone therapy for Fabry disease. Yin and Yang of enzyme inhibitors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3527132

6 July 2018

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=3527132

6 July 2018

Therapeutic approaches to protein-misfolding diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3527132

6 July 2018

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3527132

6 July 2018

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=3527132

6 July 2018

Mutant alpha-galactosidase A enzymes identified in Fabry disease patients with residual enzyme activity: biochemical characterization and restoration of normal intracellular processing by 1-deoxygalactonojirimycin

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-7-91

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/10.1186%2F1750-1172-7-91

21 January 2018

Non-invasive screening method for Fabry disease by measuring globotriaosylceramide in whole urine samples using tandem mass spectrometry

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-7-91

21 January 2018

Biomarkers of Fabry disease nephropathy

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-7-91

21 January 2018

Gb(3)/creatinine biomarkers for Fabry disease: issues to consider.

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-7-91

21 January 2018

Females with Fabry disease frequently have major organ involvement: lessons from the Fabry Registry.

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-7-91

21 January 2018

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-7-91

21 January 2018

Urinary Lipid Profiling for the Identification of Fabry Hemizygotes and Heterozygotes

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-7-91

21 January 2018

Liquid chromatography-tandem mass spectrometry quantification of globotriaosylceramide in plasma for long-term monitoring of Fabry patients treated with enzyme replacement therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3527132

28 October 2018

Chaperones: keeping a close eye on protein folding

1 reference

https://pubmed.ncbi.nlm.nih.gov/23176611

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Anderson-Fabry disease: clinical manifestations of disease in female heterozygotes

1 reference

https://pubmed.ncbi.nlm.nih.gov/23176611

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/23176611

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Agalsidase-beta therapy for advanced Fabry disease: a randomized trial

1 reference

https://pubmed.ncbi.nlm.nih.gov/23176611

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Folding of Secretory and Membrane Proteins

1 reference

https://pubmed.ncbi.nlm.nih.gov/23176611

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Differential assay for lysosomal alpha-galactosidases in human tissues and its application to Fabry's disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/23176611

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1186/1750-1172-7-91

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

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0 references

release_lypi5sh5gfcdpfdc3ga4lhs7hm

1 reference

https://api.fatcat.wiki/v0/release/lypi5sh5gfcdpfdc3ga4lhs7hm

24 November 2022

based on heuristic

mapped directly with Wikidata item

PMC publication ID

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

ResearchGate publication ID

0 references

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