- RecruitingPHASE1, PHASE2
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This study looks at a new gene therapy, SGT-003, for Duchenne muscular dystrophy. We want to see if it's safe and how well it works. Boys aged 0-17 with Duchenne can take part. It involves a single treatment and long-term follow-up.
For: Duchenne Muscular DystrophyUnited States · Canada · Italy - RecruitingPHASE3
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
This study is testing a new gene therapy, SGT-003, for boys aged 7-11 with Duchenne Muscular Dystrophy who can still walk. It's comparing SGT-003 to a dummy treatment (placebo) to see if it helps and is safe. Participants will be monitored for at least 5 years.
For: Duchenne Muscular DystrophyUnited States · Australia · Canada