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

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

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

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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