- 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
2024
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.
bioRxiv : the preprint server for biology
鈥�2024
Neurological glycogen storage diseases and emerging therapeutics.
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
鈥�2024
Perspectives of the Friedreich ataxia community on gene therapy clinical trials.
Molecular therapy. Methods & clinical development
鈥�2024
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
鈥�2024
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.
Journal of patient-reported outcomes
鈥�