Also known as: FA
Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.
Rocket Pharmaceuticals will participate in two investor conferences in March 2026, with the CEO taking part in a fireside chat and management hosting investor meetings at both events.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07005297 |
Clinical Genetics Branch Eligibility Screening Survey |
NOT_YET_RECRUITING | |
| NCT06744283 |
Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia |
RECRUITING |
