- Investigator
- Barry J Byrne
- Status
- Accepting Candidates
Research at a glance
Top areas of exploration
- Genetic Therapy , 88 publications
- Genetic Vectors , 80 publications
- Glycogen Storage Disease Type II , 61 publications
- Muscle, Skeletal , 42 publications
Research activity
Active clinical trials
This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
- Investigator
- Barry J Byrne
- Status
- Accepting Candidates
- Ages
- 7 Years - N/A
- Sexes
- Male
This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.
- Investigator
- Barry J Byrne
- Status
- Accepting Candidates
- Ages
- 18 Years - N/A
- Sexes
- All
My publications
Filter publications
361 publications
2023
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Science translational medicine
鈥�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�2023
Elevated liver glycogenolysis mediates higher blood glucose during acute exercise in Barth syndrome.
PloS one
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