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

2019

AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice.

Molecular therapy. Methods & clinical development

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2019

AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome.

Human gene therapy

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2019

AAV9-TAZ Gene Replacement Ameliorates Cardiac TMT Proteomic Profiles in a Mouse Model of Barth Syndrome.

Molecular therapy. Methods & clinical development

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2019

Blunted fat oxidation upon submaximal exercise is partially compensated by enhanced glucose metabolism in children, adolescents, and young adults with Barth syndrome.

Journal of inherited metabolic disease

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2019

Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial.

Neurology

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Research at a glance

Top areas of exploration

Research activity

Active clinical trials

My publications

Filter publications

AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice.

AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome.

AAV9-TAZ Gene Replacement Ameliorates Cardiac TMT Proteomic Profiles in a Mouse Model of Barth Syndrome.

Blunted fat oxidation upon submaximal exercise is partially compensated by enhanced glucose metabolism in children, adolescents, and young adults with Barth syndrome.

Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial.