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

2023

Optogenetic activation of the tongue in spontaneously breathing mice.

Respiratory physiology & neurobiology

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2023

Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec.

Sleep medicine

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2022

A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.

Journal of neuromuscular diseases

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2022

Addressing the implementation gap in advanced therapeutics for spinal muscular atrophy in the era of newborn screening programs.

Frontiers in neurology

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2022

Advanced therapeutic strategy for hereditary neuromuscular diseases.

Molecular therapy : the journal of the American Society of Gene Therapy

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Research at a glance

Top areas of exploration

Research activity

Active clinical trials

My publications

Filter publications

Optogenetic activation of the tongue in spontaneously breathing mice.

Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec.

A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.

Addressing the implementation gap in advanced therapeutics for spinal muscular atrophy in the era of newborn screening programs.

Advanced therapeutic strategy for hereditary neuromuscular diseases.