Sarepta Therapeutics Announces Topline Results for Part 1 of Study 102 Evaluating SRP9001 its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy 1721  Study met the primary biological endpoint of microdystrophin protein expression at 12 weeks posttreatment as measured by western blot in SRP9001treated participants versus placebo   SRP9001treated 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 posttreatment   In the prespecified analysis by agegroup by which the randomization was stratified participants aged 45 years at time of treatment with SRP9001 demonstrated a statistically significant improvement in NSAA total score versus the agematched placebo cohort achieving a 43point improvement on NSAA at 48 weeks posttreatment from baseline   No new safety signals identified for SRP9001 reinforcing the favorable safety profile observed to date   Sarepta to host conference call at 430 pm Eastern time  CAMBRIDGE Mass Jan 07 2021 GLOBE NEWSWIRE  Sarepta Therapeutics Inc NASDAQSRPT the leader in precision genetic medicine for rare diseases today announced topline results from Part 1 of Study SRP9001102 Study 102 an ongoing randomized doubleblind placebo controlled clinical trial to evaluate the safety efficacy and tolerability of a single dose of SRP9001 rAAVrh74MHCK7microdystrophin in 41 patients with Duchenne muscular dystrophy