Barry J Byrne, MD, PhD - Research

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

A Study to Evaluate the PK, Safety, Efficacy, and PD With ATB200/AT2221 in LOPD Subjects

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-na茂ve pediatric subjects with Pompe鈥�

Investigator: Barry J Byrne
Status: Accepting Candidates
Ages: 0 Years - 17 Years
Sexes: All

My publications

368 publications

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

2023

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

Science Translational Medicine

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

鈥�

2023

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

Molecular therapy. Methods & clinical development

鈥�

2023

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

Frontiers in rehabilitation sciences

鈥�

网红黑料

Research at a glance

Top areas of exploration

Research activity

Active clinical trials

My publications

Filter publications

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

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

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

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

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