Barry J Byrne, MD, PhD - Research

Research at a glance

Top areas of exploration

Genetic Therapy , 86 publications
Genetic Vectors , 80 publications
Glycogen Storage Disease Type II , 60 publications
Muscle, Skeletal , 41 publications

Research activity

368 publications

22,468 citations

Why is this important?

Active clinical trials

ATA-200 gene therapy trial in patients with LGMDR5

The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5). Patients will be treated sequentially in 2鈥�

Investigator: Barry J Byrne
Status: Accepting Candidates
Ages: 6 Years - 13 Years
Sexes: All

Pfizer Early Stage GT

The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

Investigator: Barry J Byrne
Status: Accepting Candidates
Ages: 2 Years - 3 Years
Sexes: Male

ATB200-08

This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-na茂ve pediatric subjects with IOPD.

Investigator: Barry J Byrne
Status: Accepting Candidates
Ages: N/A - 17 Years
Sexes: All

My publications

368 publications

2021

Myocardial glucose and fatty acid metabolism is altered and associated with lower cardiac function in young adults with Barth syndrome.

Journal of nuclear cardiology : official publication of the American Society of Nuclear Cardiology

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2021

NaCl and KCl mediate log increase in AAV vector particles and infectious titers in a specific/timely manner with the HSV platform.

Molecular therapy. Methods & clinical development

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2021

Phenotypic implications of pathogenic variant types in Pompe disease.

Journal of human genetics

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2021

Resistance exercise training with protein supplementation improves skeletal muscle strength and improves quality of life in late adolescents and young adults with Barth syndrome: A pilot study.

JIMD reports

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2021

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.

The Lancet. Neurology

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Research at a glance

Top areas of exploration

Research activity

Active clinical trials

My publications

Filter publications

Myocardial glucose and fatty acid metabolism is altered and associated with lower cardiac function in young adults with Barth syndrome.

NaCl and KCl mediate log increase in AAV vector particles and infectious titers in a specific/timely manner with the HSV platform.

Phenotypic implications of pathogenic variant types in Pompe disease.

Resistance exercise training with protein supplementation improves skeletal muscle strength and improves quality of life in late adolescents and young adults with Barth syndrome: A pilot study.

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.