Sarepta therapeutics announces top-line results for part 1 of study 102 evaluating srp-9001, its investigational gene therapy for the treatment of duchenne muscular dystrophy

-- study met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, in srp-9001-treated participants versus placebo -- -- srp-9001-treated participants showed an increase in nsaa total score compared to placebo at 48 weeks; however, the study did not achieve statistical significance on the primary functional endpoint of improvement in nsaa total score compared to placebo at 48 weeks post-treatment --
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