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

2024

Findings from the Longitudinal CINRG Becker Natural History Study.

Journal of neuromuscular diseases

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2024

Innate Immune Sensing of Adeno-Associated Virus Vectors.

Human gene therapy

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2024

Kenneth I. Berns, MD, PhD [1938-2024].

Human gene therapy

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2024

Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)

Journal of Neurology

2024

Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies

Pediatric Pulmonology

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Research at a glance

Top areas of exploration

Research activity

Active clinical trials

My publications

Filter publications

Findings from the Longitudinal CINRG Becker Natural History Study.

Innate Immune Sensing of Adeno-Associated Virus Vectors.

Kenneth I. Berns, MD, PhD [1938-2024].

Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)

Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies