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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 , 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

2023

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.

Science translational medicine

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2023

Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy.

Molecular therapy : the journal of the American Society of Gene Therapy

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2023

Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.

Molecular therapy. Methods & clinical development

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2023

Diaphragm pacing and independent breathing in individuals with severe Pompe disease.

Frontiers in rehabilitation sciences

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2023

Elevated liver glycogenolysis mediates higher blood glucose during acute exercise in Barth syndrome.

PloS one

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