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

2024

Meeting Report: 2023 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Therapy of Neuromuscular Diseases'.

Journal of neuromuscular diseases

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2024

Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.

bioRxiv : the preprint server for biology

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2024

Neurological glycogen storage diseases and emerging therapeutics.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics

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2024

Perspectives of the Friedreich ataxia community on gene therapy clinical trials.

Molecular therapy. Methods & clinical development

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2024

Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, 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

Meeting Report: 2023 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Therapy of Neuromuscular Diseases'.

Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.

Neurological glycogen storage diseases and emerging therapeutics.

Perspectives of the Friedreich ataxia community on gene therapy clinical trials.

Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.