Shots:

• The ongoing study involves assessing SRP-9003 (IV- with a one-time infusion) in children aged 4-15yrs. with LGMD2E. The study has cohort 1 (low dose-dose cohort- 1.85×1013 vg/kg) and cohort 2 (high-dose cohort- 7.41×1013 vg/kg)- post-treatment biopsies were taken @60days
• The cohort 1 @24mos. showed a mean beta-SG expression (54% vs 36%); IF positive fibers (48% vs 51%); mean intensity of transduced beta-SG (35% vs 47%); mean NSAD improvement of 5.7 points @18mos.; improvement across all functional measures
• The cohort 2 @ 12mos showed mean NSAD improvement (4.0 points @1yrs. vs 3.7 @6mos.). Results in both cohorts continue to reinforce the safety & tolerability profile of SRP-9003

 ­ Ref: Sarepta | Image: BioSpace