Sarepta Therapeutics Report Results of SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E

March 19, 2021 Auto Bot Clinical Trials, Report, results, Sarepta Therapeutics, Treatment 0

Shots:

  • The ongoing study involves assessing SRP-9003 (IV, with a one-time infusion) in children aged 4-15yrs. with LGMD2E. The study has cohort 1 (low dose-dose cohort, 1.85×1013 vg/kg) and cohort 2 (high-dose cohort, 7.41×1013 vg/kg), post-treatment biopsies were taken @60days
  • The cohort 1 @24mos. showed a mean beta-SG expression (54% vs 36%); IF positive fibers (48% vs 51%); mean intensity of transduced beta-SG (35% vs 47%); mean NSAD improvement of 5.7 points @18mos.; improvement across all functional measures
  • The cohort 2 @ 12mos showed mean NSAD improvement (4.0 points @1yrs. vs 3.7 @6mos.). Results in both cohorts continue to reinforce the safety & tolerability profile of SRP-9003

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

The post Sarepta Therapeutics Report Results of SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E first appeared on PharmaShots.