Barry J Byrne, MD, PhD - Research

Research at a glance

Top areas of exploration

Genetic Therapy , 88 publications
Genetic Vectors , 80 publications
Glycogen Storage Disease Type II , 61 publications
Muscle, Skeletal , 42 publications

Research activity

361 publications

22,175 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

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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2024

Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.

Journal of patient-reported outcomes

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BOB体育

Research at a glance

Top areas of exploration

Research activity

Active clinical trials

My publications

Filter publications

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.

Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.