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 , 86 publications
- Genetic Vectors , 80 publications
- Glycogen Storage Disease Type II , 60 publications
- Muscle, Skeletal , 41 publications
Research activity
Active clinical trials
- Investigator
- Barry J Byrne
- Status
- Accepting Candidates
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
368 publications
2020
Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases.
Brain sciences
•2020
Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease.
Molecular therapy. Methods & clinical development
•2020
Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis.
Archives of disease in childhood
•2020
Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start.
Journal of neurology
•2020
The CINRG Becker Natural History Study: Baseline characteristics.
Muscle & nerve
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