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
2021
Considerations for Systemic Use of Gene Therapy.
Molecular therapy : the journal of the American Society of Gene Therapy
•2021
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Molecular therapy : the journal of the American Society of Gene Therapy
•2021
Gene delivery to the hypoglossal motor system: preclinical studies and translational potential.
Gene therapy
•2021
Mavis Agbandje-McKenna's lifelong commitment to teaching and research
Molecular Therapy
•2021
Mavis Agbandje-McKenna's Lifelong Commitment to Teaching and Research.
Human gene therapy
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