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Sarepta said data presented at the World Muscle Society congress showed clinically meaningful functional benefit in ambulatory Duchenne patients aged 8 to 12 years. The company also said younger patients aged 2 to under 3 showed robust dystrophin expression and no new safety signals were identified.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today presented new data at the 31st Annual Congress of the World Muscle Society (WMS) that provide evidence of clinically meaningful functional benefit of ELEVIDYS (delandistrogene moxeparvovec) i…