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

Journal of medical genetics, 2017-04, Vol.54 (4), p.288-296

2017

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Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

Genetics in medicine, 2019-09, Vol.21 (9), p.1987-1997

2019

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Evaluating plant-soil feedback together with competition in a serpentine grassland

Ecology letters, 2007-05, Vol.10 (5), p.394-400

2007

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Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment

Journal of medical genetics, 2017-11, Vol.54 (11), p.781-786

2017

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Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial

Orphanet journal of rare diseases, 2018-04, Vol.13 (1), p.68-68, Article 68

2018

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Low frequency of Fabry disease in patients with common heart disease

Genetics in medicine, 2018-07, Vol.20 (7), p.754-759

2018

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Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study

Clinical and experimental nephrology, 2020-02, Vol.24 (2), p.157-166

2020

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Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

Journal of patient-reported outcomes, 2024-01, Vol.8 (1), p.13-12, Article 13

2024

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

Human mutation, 2011-08, Vol.32 (8), p.965-977

2011

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Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study

American journal of medical genetics. Part A, 2019-06, Vol.179 (6), p.1069-1073

2019

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Intraspecific AM Fungal Variation Contributes to Plant-Fungal Feedback in a Serpentine Grassland

Ecology (Durham), 2003-02, Vol.84 (2), p.323-336

2003

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The Herbivory Uncertainty Principle: Visiting Plants Can Alter Herbivory

Ecology (Durham), 2001-02, Vol.82 (2), p.307-312

2001

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Comparison of integrated white blood cell alpha-galactosidase a activity exposure between every-other-day orally administered migalastat and biweekly infusions of agalsidase beta or agalsidase alfa

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S63-S63

2016

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Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107971, Article 107971

2024

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SP002CLINICAL OUTCOMES WITH MIGALASTAT IN PATIENTS WITH FABRY DISEASE BASED ON DEGREE OF RENAL IMPAIRMENT: RESULTS FROM PHASE 3 TRIALS

Nephrology, dialysis, transplantation, 2018-05, Vol.33 (suppl_1), p.i346-i346

2018

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Ten years of migalastat treatment in a patient with Fabry disease: A case report

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S138-S138

2018

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Separate effects of human visitation and touch on plant growth and herbivory in an old-field community

American journal of botany, 2002-09, Vol.89 (9), p.1401-1409

2002

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SP004EFFECTS OF LONG-TERM MIGALASTAT TREATMENT ON RENAL FUNCTION BY BASELINE PROTEINURIA IN PATIENTS (PTS) WITH FABRY DISEASE

Nephrology, dialysis, transplantation, 2018-05, Vol.33 (suppl_1), p.i347-i348

2018

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Migalastat exposures in Japanese healthy volunteers and non-Japanese subjects provide evidence that they are similar to Japanese patients with Fabry disease

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S71-S72

2017

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MO068EFFICACY AND SAFETY OF MIGALASTAT, AN ORAL PHARMACOLOGICAL CHAPERONE FOR FABRY DISEASE: RENAL FINDINGS FROM TWO RANDOMIZED PHASE 3 STUDIES (FACETS AND ATTRACT)

Nephrology, dialysis, transplantation, 2017-05, Vol.32 (suppl_3), p.iii75-iii75

2017

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Effects of treatment with migalastat on the combined endpoint of kidney globotriaosylcermide accumulation and diarrhea in patients with Fabry disease: results from the phase 3 FACETS study

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S52-S52

2017

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Phenotype of Fabry disease in patients with mutations amenable to migalastat

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S58-S59

2016

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Efficacy and safety of migalastat, an oral pharmacologic chaperone for Fabry disease: results from two randomized phase 3 studies, FACETS and ATTRACT

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S45-S46

2017

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Migalastat improves diarrhea in patients with Fabry disease: results from the FACETS double-blind, placebo-controlled phase 3 study

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S119-S119

2017

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Response of patients with Fabry disease with the amenable GLA mutation p.N215S to treatment with migalastat

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S68-S69

2017

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

Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

Evaluating plant-soil feedback together with competition in a serpentine grassland

Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment

Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial

Low frequency of Fabry disease in patients with common heart disease

Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

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

Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study

Intraspecific AM Fungal Variation Contributes to Plant-Fungal Feedback in a Serpentine Grassland

The Herbivory Uncertainty Principle: Visiting Plants Can Alter Herbivory

Comparison of integrated white blood cell alpha-galactosidase a activity exposure between every-other-day orally administered migalastat and biweekly infusions of agalsidase beta or agalsidase alfa

Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL

SP002CLINICAL OUTCOMES WITH MIGALASTAT IN PATIENTS WITH FABRY DISEASE BASED ON DEGREE OF RENAL IMPAIRMENT: RESULTS FROM PHASE 3 TRIALS

Ten years of migalastat treatment in a patient with Fabry disease: A case report

Separate effects of human visitation and touch on plant growth and herbivory in an old-field community

SP004EFFECTS OF LONG-TERM MIGALASTAT TREATMENT ON RENAL FUNCTION BY BASELINE PROTEINURIA IN PATIENTS (PTS) WITH FABRY DISEASE

Migalastat exposures in Japanese healthy volunteers and non-Japanese subjects provide evidence that they are similar to Japanese patients with Fabry disease

MO068EFFICACY AND SAFETY OF MIGALASTAT, AN ORAL PHARMACOLOGICAL CHAPERONE FOR FABRY DISEASE: RENAL FINDINGS FROM TWO RANDOMIZED PHASE 3 STUDIES (FACETS AND ATTRACT)

Effects of treatment with migalastat on the combined endpoint of kidney globotriaosylcermide accumulation and diarrhea in patients with Fabry disease: results from the phase 3 FACETS study

Phenotype of Fabry disease in patients with mutations amenable to migalastat

Efficacy and safety of migalastat, an oral pharmacologic chaperone for Fabry disease: results from two randomized phase 3 studies, FACETS and ATTRACT

Migalastat improves diarrhea in patients with Fabry disease: results from the FACETS double-blind, placebo-controlled phase 3 study

Response of patients with Fabry disease with the amenable GLA mutation p.N215S to treatment with migalastat

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