Logo

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

Share this

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

Click here to­ read the full press release 

Senior Editor

This content piece was prepared by our former Senior Editor. She had expertise in life science research and was an avid reader. For any query reach out to us at connect@pharmashots.com

Share this article on WhatsApp, LinkedIn and Twitter

Join the PharmaShots family of 12000+ subscribers

I accept the Terms and Conditions