ÍøºìºÚÁÏ

Skip to main content
(352) 294-5757

Barry J Byrne, MD, PhD

Pediatric Cardiologist

Photo of Barry J Byrne
(352) 294-5757 MyUFÍøºìºÚÁÏ

Research at a glance

Top areas of exploration

  • Genetic Therapy , 86 publications
  • Genetic Vectors , 80 publications
  • Glycogen Storage Disease Type II , 60 publications
  • Muscle, Skeletal , 41 publications

Research activity

368 publications

22,468 citations

Why is this important?

Active clinical trials

ATA-200 gene therapy trial in patients with LGMDR5

The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5). Patients will be treated sequentially in 2…

Investigator
Barry J Byrne
Status
Accepting Candidates
Ages
6 Years - 13 Years
Sexes
All
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

368 publications

2019

Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy.

Annals of translational medicine

•

2019

Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy

Current Gene Therapy

2019

Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.

Neuromuscular disorders : NMD

•

2019

Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.

Human gene therapy

•

2018

A multicenter, retrospective medical record review of X-linked myotubular myopathy: The recensus study.

Muscle & nerve

•