- 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
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver.
Molecular therapy. Methods & clinical development
鈥�2024
Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.
Frontiers in neurology
鈥�2024
Codon-Optimized and de novo-Synthesized E-Selectin/AAV2 Dose-Response Study for Vascular Regeneration Gene Therapy.
Annals of surgery
鈥�2024
Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.
Frontiers in neurology
鈥�2024
Findings from the Longitudinal CINRG Becker Natural History Study.
Journal of neuromuscular diseases
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