• Programme
  • Discovery
  • Preclinical
  • Clinical
Eteplirsen (SRP-4051)
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)
CRISPR/CAS9 (Duke University)
MYO-301 (LGMD2L Anoctamin 5)
Pompe Disease
CNS-1
CNS-2
Neutrophin 3 (CMT TYPE 1A)
LYS-SAF 302 (MPS TYPE IIIA)