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Barry J Byrne, MD, PhD

Pediatric Cardiologist

Photo of Barry J Byrne

Research at a glance

Top areas of exploration

  • Genetic Therapy , 77 publications
  • Genetic Vectors , 76 publications
  • Glycogen Storage Disease Type II , 56 publications
  • Muscle, Skeletal , 40 publications

Research activity

340 publications

19,903 citations

Why is this important?

Active clinical trials

Sarepta 4658-402

This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2…

Investigator
Barry J Byrne
Status
Accepting Candidates
Ages
4 Years - 13 Years
Sexes
Male
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-naïve pediatric subjects with IOPD.

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

My publications

340 publications

2024

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

PubMed Publisher's site

2024

Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies

Pediatric Pulmonology

Publisher's site

2024

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

Molecular therapy. Methods & clinical development

PubMed Publisher's site

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

PubMed Publisher's site

2024

The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.

Neurology and therapy

PubMed Publisher's site