- Investigator
- Barry J Byrne
- Status
- Accepting Candidates
Research at a glance
Top areas of exploration
- Genetic Therapy , 77 publications
- Genetic Vectors , 76 publications
- Glycogen Storage Disease Type II , 57 publications
- Muscle, Skeletal , 40 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
339 publications
2024
Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies
Pediatric Pulmonology
Publisher's site2024
Perspectives of the Friedreich ataxia community on gene therapy clinical trials.
Molecular therapy. Methods & clinical development
PubMed • Publisher's site2024
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
The Lancet. Neurology
PubMed • Publisher's site2024
The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.
Neurology and therapy
PubMed • Publisher's site2024
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies
Journal of Clinical Investigation
Publisher's site