Sarepta Reports Two-Year Follow Up Results of SRP-9001 for Duchenne Muscular Dystrophy

 Sarepta Reports Two-Year Follow Up Results of SRP-9001 for Duchenne Muscular Dystrophy

Sarepta Reports Two-Year Follow Up Results of SRP-9001 for Duchenne Muscular Dystrophy

Shots:

  • Study 101 involves assessing of SRP-9001 (AAVrh74.MHCK7.micro-dystrophin, 2×1014 vg/kg) vs PBO in 4 patients aged 4-7yrs. with DMD
  • The study demonstrated a mean 7.0 point improvement in NSSA score, therapy was well tolerated over 2yrs. time period, @90days. patients had confirmed vector transduction, reduction in CK level
  • SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue, targeting the production of the micro-dystrophin protein. In Dec’2019, Sarepta granted Roche the exclusive right to launch and commercialize SRP-9001 outside the US

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

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