Shots:

• The Study 102 involves assessing of SRP-9001 vs PBO in 41 patients in a ratio (1:1) with DMD aged 4-7yrs.
• Results: @12wks. post treatment- the study met its 1EPs of micro-dystrophin protein expression- as measured by western blot. @48wks. increase in NSAA total score- however- the study did not achieve significance on the primary functional endpoint of improvement in NSAA total score @48wks. post-treatment
• The pre-specified analysis of patients aged 4-5yrs. showed improvement in NSAA total score (4.3 vs 1.9 points). No new safety signals identified for SRP-9001- reinforcing the favorable safety profile

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