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
2023
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation
Molecular Therapy - Methods & Clinical Development
2023
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy
Science Translational Medicine
2023
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy.
Molecular therapy : the journal of the American Society of Gene Therapy
•2023
Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.
Molecular therapy. Methods & clinical development
•2023
Diaphragm pacing and independent breathing in individuals with severe Pompe disease.
Frontiers in rehabilitation sciences
•