Shots:

• Study 101 involves assessing of SRP-9001 (AAVrh74.MHCK7.micro-dystrophin- 2x1014 vg/kg) vs PBO in 4 patients aged 4-7yrs. with DMD
• The study demonstrated a mean 7.0 point improvement in NSSA score- therapy was well tolerated over 2yrs. time period- @90days. patients had confirmed vector transduction- reduction in CK level
• SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue- targeting the production of the micro-dystrophin protein. In Dec’2019- Sarepta granted Roche the exclusive right to launch and commercialize SRP-9001 outside the US

­ Ref: Sarepta | Image: Sarepta