Sarepta Therapeutics Announces Positive Functional Results from the SRP-9003 (MYO-101) Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E, or Beta-Sarcoglycanopathy
Sarepta Therapeutics, Inc. (SRPT)
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Source: GlobeNewswire
-- Improvements on functional measures seen in all three participants -- -- Significant reduction in creatine kinase maintained over nine months -- -- Results follow positive and robust expression and biomarker data presented earlier in 2019 -- CAMBRIDGE, Mass., Oct. 04, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the nine-month functional results from three Limb-girdle muscular dystrophy Type 2E (LGMD2E) clinical trial participants who received SRP-9003. SRP-9003 is an investigational gene therapy intended to transduce skeletal and cardiac muscle with a gene that codes for the full-length, native beta-sarcoglycan protein, the lack of which is the sole cause of LGMD2E. In Cohort 1 of the SRP-9003 study, three participants ages 4-13 were treated with an infusion of SRP-9003 at a dose of 5x1013vg/kg. Improvements in functional outcomes were observed at day 270 (nine months) for all t
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