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

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