Duchenne muscular dystrophy
Gene therapy, exon skipping, and cell therapies racing toward broader DMD labels.
Lenses
EarlierApproval →
SRPTApprovedcash ok
2026-11-15 (94d)Company-guided
Elevidys microdystrophin · NCT04626674 · Label / expansion update
CAPRFiledcash ok
2026-08-22 (9d)Confirmed
Deramiocel cell therapy · NCT05126758 · PDUFA — deramiocel DMD
RCKTPh2cash ok
2026-12-01 (110d)Estimated
Gene therapy program · NCT05775848 · Phase 2 update
SLDBPh1cash ok
2027-03-01 (200d)Estimated
SGT-003 microdystrophin · NCT06138639 · Dose expansion data