CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the U.S. Food & ...
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. We have received feedback from the Medicines & Healthcare products Regulatory Agency (MHRA) in th...
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported new results from Study 9001-103. Also known as ENDEAVOR, Study 9001-103 is a multi-cohort study of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of Duchenne muscular dystrophy. Treatment with ELEVIDYS in the ENDEAVOR participant...
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today presented new data from Part 2 of the EMBARK study that continue to support the clinical benefits of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. These data are among other EL...
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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the Japanese Ministry of Health, Labour, and Welfare (MHLW) has approved ELEVIDYS (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy (DMD) under the conditional and time-limited approval pathway in Japan. ELEVI...
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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will participate in a fireside chat at the BofA Securities Health Care Conference at the Encore Hotel in Las Vegas, Nev. on Wednesday, May 14, 2025 at 8:40 a.m. P.T./11:40 a.m. E.T. The presentations will be webcast live ...
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