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

2025

Effect of Tadalafil on cardiac function and left ventricular dimensions in Duchenne muscular dystrophy: safety and cardiac MRI substudy results from a randomized, placebo-controlled trial.

BMC cardiovascular disorders

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2025

Emerging therapies in hereditary ataxias.

Trends in molecular medicine

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2025

Overexpression of Wild-Type TMEM43 Improves Cardiac Function in Arrhythmogenic Right Ventricular Cardiomyopathy Type 5

Circulation Research

2025

Quantification and comparison of anti-AAV9 and anti-AAVrh74 antibodies in plasma and human milk: Implications for AAV-based gene therapy candidacy

Journal of Neuromuscular Diseases

2025

Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL.

Muscle & nerve

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网红黑料

Research at a glance

Top areas of exploration

Research activity

Active clinical trials

My publications

Filter publications

Effect of Tadalafil on cardiac function and left ventricular dimensions in Duchenne muscular dystrophy: safety and cardiac MRI substudy results from a randomized, placebo-controlled trial.

Emerging therapies in hereditary ataxias.

Overexpression of Wild-Type TMEM43 Improves Cardiac Function in Arrhythmogenic Right Ventricular Cardiomyopathy Type 5

Quantification and comparison of anti-AAV9 and anti-AAVrh74 antibodies in plasma and human milk: Implications for AAV-based gene therapy candidacy

Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL.