RAP-219’s lead program demonstrated robust and consistent activity in patients with focal onset seizures (FOS) in Phase 2a trial; initiation of Phase 3 program expected in the second quarter of 2026 ...
HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 - - 48-week trial will enroll approximately 150 individuals, and first sites are now open for ...