9001-303 (ENVISION)
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the…
- Investigator
- Carmen Leon Astudillo
- Status
- Accepting Candidates
- Ages
- N/A - N/A
- Sexes
- Male
EMBARK
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with…
- Investigator
- Carmen Leon Astudillo
- Status
- Accepting Candidates
- Ages
- 4 Years - 7 Years
- Sexes
- Male