Shots:

• Sarepta reported positive 12wks. expression & safety results from the first 11 participants enrolled in Study SRP-9001-103. The study uses commercially representative SRP-9001
• Patients demonstrated robust transduction- with a mean micro-dystrophin expression of 55.4% of normal- as measured by western blot. Micro-dystrophin was properly localized to the muscle sarcolemma- with patients achieving a mean percentage of dystrophin positive fibers of 70.5% and intensity of micro-dystrophin expression of 116.9% of normal control
• Mean vector genome copies per nucleus reached 3.87. The safety profile is consistent with prior studies and no new safety signals identified

  Ref: GLOBE NEWSWIRE | Image: Sarepta