• Program
  • Discovery
  • Preclinical
  • Phase I
  • Phase II
  • Phase III
  • Commercial*
Exondys 51 (Eteplirsen)*
Golodirsen (SRP-4053)
Casimersen (SRP-4045)
Exon 52
Other Exon Targets**
SRP-5051
SRP-5053
SRP-5045
SRP-5052
SRP-5044
SRP-5050
GALGT2 (Nationwide)
Micro-Dystrophin (Nationwide)
Micro-Dystrophin (Genethon)
MYO-101 (LGMD2E β-sarcoglycan)
MYO-102 (LGMD2D α-sarcoglycan)
MYO-103 (LGMD2C γ-sarcoglycan)
MYO-201 (LGMD2B Dysferlin)
MYO-301 (LGMD2L Anoctamin 5)
Pompe Disease
CNS-1
CNS-2
CRISPR/CAS9 (DUKE UNIVERSITY)