Barry J Byrne, MD, PhD - Research

Research at a glance

Top areas of exploration

Genetic Therapy , 88 publications
Genetic Vectors , 81 publications
Glycogen Storage Disease Type II , 61 publications
Muscle, Skeletal , 42 publications

Research activity

361 publications

21,624 citations

Why is this important?

Active clinical trials

Pfizer Early Stage GT

The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

Investigator: Barry J Byrne
Status: Accepting Candidates
Ages: 2 Years - 3 Years
Sexes: Male

ATB200-08

This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-na茂ve pediatric subjects with IOPD.

Investigator: Barry J Byrne
Status: Accepting Candidates
Ages: N/A - 17 Years
Sexes: All

TECPR2 Obs

The purpose of this study is to learn more about the disease progression in patients with a TECPR2 mutation.

Investigator: Barry J Byrne
Status: Accepting Candidates
Ages: 18 Months - 12 Years
Sexes: All

My publications

361 publications

2025

Correction: Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)

Journal of Neurology

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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

2024

104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).

Journal of neurology

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BOB体育

Research at a glance

Top areas of exploration

Research activity

Active clinical trials

My publications

Filter publications

Correction: Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)

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

104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).