Table 2 Dystrophin restoration drugs approved and in clinical development
Name | Company | Therapeutic strategies | Approval or clinical stage |
|---|---|---|---|
Eteplirsen | Sarepta Therapeutics | Exon 51 skipping | FDA |
Viltolarsen | NS Pharma | Exon 53 skipping | FDA |
Golodirsen | Sarepta Therapeutics | Exon 53 skipping | FDA, JAPAN |
Casimersen | Sarepta Therapeutics | Exon 45 skipping | FDA |
Delandistrogene moxeparvovec (SRP-9001) | Sarepta Therapeutics | Micro-dystrophin | FDA |
PF-06939926 | Pfizer | Micro-dystrophin | Phase III |
SGT-001 | Solid Biosciences | Micro-dystrophin | Phase I/II |
RGX-202 | REGENXBIO | Micro-dystrophin | Phase I/II |