Sarepta's SRP-9001 Demonstrates Robust Expression and Safety Results in ENDEAVOR Study for DMD
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
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