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

2021

Safety, feasibility, and efficacy of strengthening exercise in Duchenne muscular dystrophy.

Muscle & nerve

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2021

Scratching the surface of RGD-directed AAV capsid engineering.

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

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2020

Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking.

Viruses

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2020

Advancements in AAV-mediated Gene Therapy for Pompe Disease.

Journal of neuromuscular diseases

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2020

Arginine kinetics are altered in a pilot sample of adolescents and young adults with Barth syndrome.

Molecular genetics and metabolism reports

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网红黑料

Research at a glance

Top areas of exploration

Research activity

Active clinical trials

My publications

Filter publications

Safety, feasibility, and efficacy of strengthening exercise in Duchenne muscular dystrophy.

Scratching the surface of RGD-directed AAV capsid engineering.

Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking.

Advancements in AAV-mediated Gene Therapy for Pompe Disease.

Arginine kinetics are altered in a pilot sample of adolescents and young adults with Barth syndrome.