BOB体育

Skip to main content

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

2024

Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy.

International journal of molecular sciences

鈥�

2024

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

Neurology and therapy

鈥�

2024

The multifaceted roles of the brain glycogen.

Journal of neurochemistry

鈥�

2024

Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies

Journal of Clinical Investigation

2023

Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.

Molecular therapy. Methods & clinical development

鈥�