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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

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
Givinostat Long Term Study

This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.

Investigator
Barry J Byrne
Status
Accepting Candidates
Ages
7 Years - N/A
Sexes
Male

My publications

340 publications

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

PubMed Publisher's site

2024

B cell focused transient immune suppression protocol for efficient AAV readministration to the liver.

Molecular therapy. Methods & clinical development

PubMed Publisher's site

2024

Findings from the Longitudinal CINRG Becker Natural History Study.

Journal of neuromuscular diseases

PubMed Publisher's site

2024

In memoriam: Kenneth I. Berns, MD, PhD (1938-2024).

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

PubMed Publisher's site