- Investigator
- Barry J Byrne
- Status
- Accepting Candidates
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
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
367 publications
2025
Cardiopulmonary exercise testing as an integrative approach to explore physiological limitations in Duchenne muscular dystrophy.
Journal of neuromuscular diseases
鈥�2025
Clinical advances in gene, cell, and RNA therapies.
Molecular therapy : the journal of the American Society of Gene Therapy
鈥�2025
Correction: 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
鈥�2025
Current clinical applications of AAV-mediated gene therapy
Molecular Therapy
2025
Effect of Tadalafil on cardiac function and left ventricular dimensions in Duchenne muscular dystrophy: safety and cardiac MRI substudy results from a randomized, placebo-controlled trial.
BMC cardiovascular disorders
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