Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. (Q33886219)

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English	Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.	scientific article

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27 March 2020

Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study (English)

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27 March 2020

molecular chaperones

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enzyme replacement therapy

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based on heuristic

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Derralynn A Hughes

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Gere Sunder-Plassmann

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Dominique P Germain

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Daniel-G. Bichet

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Ulla Feldt-Rasmussen

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Ulla Feldt-Rasmussen

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Suma P Shankar

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David Koeller

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Khan Nedd

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Takashi Hamazaki

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Toya Ohashi

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Iacopo Olivotto

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Norio Sakai

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Patrick Deegan

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François Eyskens

15

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Ozlem Goker-Alpan

17

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Ana Jovanovic

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Charles M Lourenco

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Ichiei Narita

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Mark Thomas

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William R Wilcox

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Raphael Schiffmann

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Elizabeth Ludington

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Christopher Viereck

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John Kirk

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Julie Yu

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Franklin Johnson

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Pol Boudes

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Elfrida R Benjamin

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David J Lockhart

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Carrolee Barlow

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Nina Skuban

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Jeffrey P Castelli

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Jay Barth

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language of work or name

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10 November 2016

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Journal of Medical Genetics

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288-296

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4

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27 March 2020

The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat

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5 July 2018

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

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5 July 2018

Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years' treatment

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5 July 2018

Serum-Mediated Inhibition of Enzyme Replacement Therapy in Fabry Disease.

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5 July 2018

Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease

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5 July 2018

Patients with Fabry disease after enzyme replacement therapy dose reduction versus treatment switch.

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5 July 2018

Natural course of Fabry disease and the effectiveness of enzyme replacement therapy: a systematic review and meta-analysis: effectiveness of ERT in different disease stages

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5 July 2018

Fibrosis: a key feature of Fabry disease with potential therapeutic implications

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5 July 2018

Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry

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5 July 2018

Long-term outcome of enzyme-replacement therapy in advanced Fabry disease: evidence for disease progression towards serious complications

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5 July 2018

Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain

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5 July 2018

Effects of enzyme replacement therapy in adult patients with Fabry disease on cardiac structure and function: a retrospective cohort study of the Fabry Munster Study (FaMuS) data

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5 July 2018

Neonatal screening for lysosomal storage disorders: feasibility and incidence from a nationwide study in Austria

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5 July 2018

Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation

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5 July 2018

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

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5 July 2018

Cardiovascular events in patients with fabry disease natural history data from the fabry registry

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5 July 2018

The Fabry cardiomyopathy: models for the cardiologist

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5 July 2018

Prognostic indicators of renal disease progression in adults with Fabry disease: natural history data from the Fabry Registry

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5 July 2018

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

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5 July 2018

Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data

1 reference

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5 July 2018

End-stage renal disease in patients with Fabry disease: natural history data from the Fabry Registry

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5 July 2018

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

1 reference

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5 July 2018

Life expectancy and cause of death in males and females with Fabry disease: findings from the Fabry Registry

1 reference

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5 July 2018

A new equation to estimate glomerular filtration rate

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5 July 2018

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

1 reference

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5 July 2018

Agalsidase alfa and kidney dysfunction in Fabry disease.

1 reference

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5 July 2018

Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5502308

5 July 2018

Agalsidase alfa slows the decline in renal function 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=5502308

5 July 2018

Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy 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=5502308

5 July 2018

High incidence of later-onset fabry disease revealed by newborn screening

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5502308

5 July 2018

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

1 reference

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5 July 2018

Enzyme replacement and enhancement therapies for lysosomal diseases

1 reference

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5 July 2018

Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy

1 reference

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5 July 2018

The MOS 36-ltem Short-Form Health Survey (SF-36)

1 reference

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5 July 2018

Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.

1 reference

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29 October 2018

X-chromosome inactivation in female 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=5502308

29 October 2018

Cardiovascular outcomes in Fabry disease are linked to severity of chronic kidney disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5502308

29 October 2018

1 reference

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29 October 2018

Plasma globotriaosylsphingosine: diagnostic value and relation to clinical manifestations of Fabry disease

1 reference

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29 October 2018

Effect of agalsidase alfa replacement therapy on Fabry disease-related hypertrophic cardiomyopathy: a 12- to 36-month, retrospective, blinded echocardiographic pooled analysis

1 reference

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29 October 2018

Nephropathy in males and females with Fabry disease: cross-sectional description of patients before treatment with enzyme replacement therapy.

1 reference

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29 October 2018

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

1 reference

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29 October 2018

Evaluation of the modification of diet in renal disease study equation in a large diverse population.

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29 October 2018

Fabry disease: baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry.

1 reference

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29 October 2018

High Incidence of the Cardiac Variant of Fabry Disease Revealed by Newborn Screening in the Taiwan Chinese Population

1 reference

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Iohexol Clearance for the Determination of Glomerular Filtration Rate: 15 Years' Experience in Clinical Practice

1 reference

https://pubmed.ncbi.nlm.nih.gov/27834756

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

The kidney in Fabry's disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/27834756

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/27834756

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/27834756

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Multiplex tandem mass spectrometry analysis of novel plasma lyso-Gb₃-related analogues in Fabry disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/27834756

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/27834756

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Reduction of elevated plasma globotriaosylsphingosine in patients with classic Fabry disease following enzyme replacement therapy

1 reference

https://pubmed.ncbi.nlm.nih.gov/27834756

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Long-term effects of enzyme replacement therapy on fabry cardiomyopathy: evidence for a better outcome with early treatment

1 reference

https://pubmed.ncbi.nlm.nih.gov/27834756

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1136/JMEDGENET-2016-104178

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27 March 2020

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27 March 2020

1 reference

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27 March 2020

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