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

A Study to Evaluate the PK, Safety, Efficacy, and PD With ATB200/AT2221 in LOPD Subjects

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-na茂ve pediatric subjects with Pompe鈥�

Investigator: Barry J Byrne
Status: Accepting Candidates
Ages: 0 Years - 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.