• Programm
  • Entdeckung
  • Vorklinisch
  • Phase I
  • Phase II
  • Phase III
Eteplirsen (SRP-4051)
Golodirsen (SRP-4053)
Casimersen (SRP-4045)
Exon 52
Sonstige Exon-Ziele*
SRP-5051
SRP-5053
SRP-5045
SRP-5052
SRP-5044
SRP-5050
GALGT2 (Nationwide)
Mikro-Dystrophin (Nationwide)
Mikro-Dystrophin (Genethon)
MYO-101 (LGMD2E β-sarcoglycan)
MYO-102 (LGMD2D α-sarcoglycan)
MYO-103 (LGMD2C γ-sarcoglycan)
MYO-201 (LGMD2B Dysferlin)
CRISPR/CAS9 (Duke Universität)
MYO-301 (LGMD2L Anoctamin 5)
Morbus Pompe
ZNS-1
ZNS-2
Neutrophin 3 (CMT TYPE 1A)
LYS-SAF 302 (MPS TYPE IIIA)