Sarepta therapeutics' gene therapy srp-9001 shows statistically significant functional improvements compared to pre-specified matched external control in part 2 of study srp-9001-102 for the treatment of duchenne muscular dystrophy

Cambridge, mass., jan. 10, 2022 (globe newswire) -- sarepta therapeutics, inc. (nasdaq:srpt), the leader in precision genetic medicine for rare diseases, today announced topline results from part 2 of study srp-9001-102 (study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of srp-9001 (delandistrogene moxeparvovec) in 41 patients with duchenne muscular dystrophy, 21 of whom were in the placebo crossover cohort. srp-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. results were presented today at the 40th annual j.p. morgan healthcare conference.
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