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

Considerations for Systemic Use of Gene Therapy.

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

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2021

Current Clinical Applications of In聽Vivo Gene Therapy with AAVs.

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

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2021

Gene delivery to the hypoglossal motor system: preclinical studies and translational potential.

Gene therapy

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2021

Mavis Agbandje-McKenna's lifelong commitment to teaching and research

Molecular Therapy

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2021

Mavis Agbandje-McKenna's Lifelong Commitment to Teaching and Research.

Human gene therapy

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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

Considerations for Systemic Use of Gene Therapy.

Current Clinical Applications of In聽Vivo Gene Therapy with AAVs.

Gene delivery to the hypoglossal motor system: preclinical studies and translational potential.

Mavis Agbandje-McKenna's lifelong commitment to teaching and research

Mavis Agbandje-McKenna's Lifelong Commitment to Teaching and Research.