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Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia

Human gene therapy, 2023-07, Vol.34 (13-14), p.605-615

2023

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Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study

Molecular genetics and metabolism, 2020-09, Vol.131 (1-2), p.219-228

2020

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THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

Muscle & nerve, 2013-09, Vol.48 (3), p.343-356

2013

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The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study

Muscle & nerve, 2013-09, Vol.48 (3), p.357-368

2013

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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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FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument

Quality of life research, 2021-10, Vol.30 (10), p.2983-2994

2021

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Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy

PloS one, 2013-12, Vol.8 (12), p.e81302-e81302

2013

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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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Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study)

Orphanet journal of rare diseases, 2020-06, Vol.15 (1), p.158-158, Article 158

2020

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Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study

Orphanet journal of rare diseases, 2020-07, Vol.15 (1), p.1-190, Article 190

2020

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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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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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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

PloS one, 2015-08, Vol.10 (8), p.e0134341-e0134341

2015

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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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Planting in Fertile Soil: The National Rifle Association and State Firearms Legislation

Social science quarterly, 2017-06, Vol.98 (2), p.485-499

2017

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Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report

Case reports in obstetrics and gynecology, 2019, Vol.2019 (2019), p.1-7

2019

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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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Direct Democracy, Educative Effects, and the (Mis)Measurement of Ballot Measure Awareness

Political behavior, 2020-12, Vol.42 (4), p.1015-1034

2020

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Arkansas's Fight for Real Equity

State Education Standard, 2015-05, Vol.15 (2), p.21

Barth, Jay

2015

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

Genetics in medicine, 2017-04, Vol.19 (4), p.430-438

2017

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Abstract 13599: LX2020, an Adeno Associated Viral-Based Plakophilin 2 Gene Therapy Stabilizes Cardiac Disease Phenotype in a Severe Mouse Model of Arrhythmogenic Right Ventricular Cardiomyopathy

Circulation (New York, N.Y.), 2022-11, Vol.146 (Suppl_1)

2022

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Effect of long-term migalastat treatment on plasma globotriaosylsphingosine (lyso-Gb3) levels in patients with Fabry disease previously treated with enzyme replacement therapy: Results from ATTRACT and open-label extension studies

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S31-S31

2019

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Migalastat pharmacokinetic (PK) exposure comparisons between race/ethnic groups and between males and females are similar

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S80-S81

2019

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Educating Citizens or Defying Federal Authority? A Comparative Study of In-State Tuition for Undocumented Students

Policy studies journal, 2010-08, Vol.38 (3), p.419-445

2010

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Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia

Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study

THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter 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

FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument

Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy

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

Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study)

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial

Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study

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

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

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

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

Planting in Fertile Soil: The National Rifle Association and State Firearms Legislation

Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report

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

Direct Democracy, Educative Effects, and the (Mis)Measurement of Ballot Measure Awareness

Arkansas's Fight for Real Equity

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

Abstract 13599: LX2020, an Adeno Associated Viral-Based Plakophilin 2 Gene Therapy Stabilizes Cardiac Disease Phenotype in a Severe Mouse Model of Arrhythmogenic Right Ventricular Cardiomyopathy

Effect of long-term migalastat treatment on plasma globotriaosylsphingosine (lyso-Gb3) levels in patients with Fabry disease previously treated with enzyme replacement therapy: Results from ATTRACT and open-label extension studies

Migalastat pharmacokinetic (PK) exposure comparisons between race/ethnic groups and between males and females are similar

Educating Citizens or Defying Federal Authority? A Comparative Study of In-State Tuition for Undocumented Students

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