• Programma
  • Scoperta
  • Preclinica
  • Clinico
Eteplirsen (SRP-4051)
Golodirsen (SRP-4053)
Casimersen (SRP-4045)
Essone 52
Altri Essoni Target*
SRP-5051
SRP-5053
SRP-5045
SRP-5052
SRP-5044
SRP-5050
GALGT2 (Nationwide)
Micro-Distrofina (Nationwide)
Micro-Distrofina (Genethon)
MYO-101 (LGMD2E β-sarcoglicano)
MYO-102 (LGMD2D α-sarcoglicano)
MYO-103 (LGMD2C γ-sarcoglycan)
MYO-201 (LGMD2B Disferlina)
CRISPR/CAS9 (Duke University)
MYO-301 (LGMD2L Anocatamina 5)
Malattia di Pompe
SNC-1
SNC-2
Neutrophin 3 (CMT TYPE 1A)
LYS-SAF 302 (MPS TYPE IIIA)