Drug Name | Type of Drug | DMD Mutations Addressed | Sponsor | Current Development Phase | Approval Status |
|---|---|---|---|---|---|
PF-06939926 | IV infusion given once only | All with the exception of exons 9-13 or deletion effecting both 29 or 30 | Pfizer | Just started Phase 3 | Still under investigation |
SRP-9001 | IV infusion given once only | All with the exception of exons 1-17 and exon 45 | Sarepta | Starting Phase 3 | Still under investigation |
GALGT2 | IV infusion given once only | All | Nationwide Children’s Hospital (USA only) | Phase 1 and 2 | Still under investigation |
SGT-001 | IV infusion given once only | All | Solid Biosciences | Phase 1 and 2 | Still under investigation |
GNT 0004 | IV infusion given once only | All but may change soon | Genethon | Phase 1/2/3 but trial currently suspended due to serious adverse events | Still under investigation |