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

 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

Click here to­ read full press release/ article | Ref: GLOBE NEWSWIRE | Image: Sarepta

Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

Related post