Shots:

• The SRP-9001 program consists of 4 studies i.e.- SRP-9001-301/103/102/101 that evaluates SRP-9001 in patients with DMD
• In studies 101 & 102- SRP-9001-treated patients showed improvement in 8.6 & 2.9 points on NSAA over natural history cohort @3yrs. & 1yrs. after treatment & functional results from 103 study (cohort 1) showed 3.0 points improvement from baseline on NSAA @6mos.- safety & tolerability is similar to previous reports
• The company also presents the details of the P-III SRP-9001-301 (EMBARK) trial evaluates SRP-9001 vs PBO in 120 patients with DMD & therapy is being developed in collaboration with Roche. The 1EPs will assess the change in NSAA total score from baseline @52wks.

  | Ref: Sarepta | Image: BioSpace