The purpose of this study is to learn more about the disease progression in patients with a TECPR2 mutation.
- Investigator
- Barry J Byrne
- Status
- Accepting Candidates
- Ages
- 18 Months - 12 Years
- Sexes
- All
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
- Investigator
- Barry J Byrne
- Status
- Accepting Candidates
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
My publications
Filter publications
339 publications
2024
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
Journal of neurology
PubMed • Publisher's site2024
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver.
Molecular therapy. Methods & clinical development
PubMed • Publisher's site2024
Findings from the Longitudinal CINRG Becker Natural History Study.
Journal of neuromuscular diseases
PubMed • Publisher's site2024
Kenneth I. Berns, MD, PhD [1938-2024].
Human gene therapy
PubMed • Publisher's site2024
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
PubMed • Publisher's site