(Q33886219)

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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scholarly 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

0 references

enzyme replacement therapy

0 references

1 reference

based on heuristic

inferred from title

Derralynn A Hughes

1

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27 March 2020

2

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27 March 2020

Gere Sunder-Plassmann

4

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27 March 2020

7

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27 March 2020

9

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27 March 2020

14

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27 March 2020

Dominique P Germain

16

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27 March 2020

18

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27 March 2020

Daniel-G. Bichet

24

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27 March 2020

Ulla Feldt-Rasmussen

38

object named as

Ulla Feldt-Rasmussen

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27 March 2020

author name string

Suma P Shankar

3

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27 March 2020

David Koeller

5

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27 March 2020

Khan Nedd

6

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27 March 2020

Takashi Hamazaki

8

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27 March 2020

Toya Ohashi

10

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27 March 2020

Iacopo Olivotto

11

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27 March 2020

Norio Sakai

12

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27 March 2020

Patrick Deegan

13

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27 March 2020

François Eyskens

15

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27 March 2020

Ozlem Goker-Alpan

17

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27 March 2020

Ana Jovanovic

19

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27 March 2020

Charles M Lourenco

20

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27 March 2020

Ichiei Narita

21

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27 March 2020

Mark Thomas

22

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27 March 2020

William R Wilcox

23

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27 March 2020

Raphael Schiffmann

25

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27 March 2020

Elizabeth Ludington

26

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27 March 2020

Christopher Viereck

27

1 reference

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27 March 2020

John Kirk

28

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27 March 2020

Julie Yu

29

1 reference

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27 March 2020

Franklin Johnson

30

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27 March 2020

Pol Boudes

31

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27 March 2020

Elfrida R Benjamin

32

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27 March 2020

David J Lockhart

33

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27 March 2020

Carrolee Barlow

34

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27 March 2020

Nina Skuban

35

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27 March 2020

Jeffrey P Castelli

36

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27 March 2020

Jay Barth

37

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27 March 2020

language of work or name

0 references

publication date

10 November 2016

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27 March 2020

Journal of Medical Genetics

1 reference

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27 March 2020

54

1 reference

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27 March 2020

288-296

1 reference

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27 March 2020

4

1 reference

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27 March 2020

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

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

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=5502308

5 July 2018

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

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

Serum-Mediated Inhibition of Enzyme Replacement Therapy in 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

Ten-year outcome of enzyme replacement therapy with agalsidase beta 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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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 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=5502308

5 July 2018

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

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

The Fabry cardiomyopathy: models for the cardiologist

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

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

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

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=5502308

5 July 2018

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

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

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

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

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=5502308

5 July 2018

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

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 new equation to estimate glomerular filtration rate

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

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=5502308

5 July 2018

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=5502308

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

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

5 July 2018

Enzyme replacement and enhancement therapies for lysosomal diseases

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

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

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

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

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

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

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

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

29 October 2018

Plasma globotriaosylsphingosine: diagnostic value and relation to clinical manifestations of 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

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

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

Nephropathy in males and females with Fabry disease: cross-sectional description of patients before treatment 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=5502308

29 October 2018

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

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

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

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

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

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

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

1 reference

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

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

1 reference

Europe PubMed Central

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27834756%20AND%20SRC:MED&resulttype=core&format=json

27 March 2020

1 reference

Europe PubMed Central

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27834756%20AND%20SRC:MED&resulttype=core&format=json

27 March 2020

1 reference

Europe PubMed Central

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27834756%20AND%20SRC:MED&resulttype=core&format=json

27 March 2020

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