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As an early investor in Dyne Therapeutics, CureDuchenne is pleased to share that Dyne announced positive data from their Phase 1/2 trial of DYNE-251 in individuals with #Duchenne amenable to skipping exon 51. Individuals treated with 20 mg/kg once every 4 weeks for 6 months showed a mean dystrophin expression of 3.7% of normal when unadjusted for muscle content; when adjusted for muscle contest, the mean was 8.72% of normal dystrophin. Improvements were observed in multiple functional endpoints in the 20 mg/kg cohort at 6 months, as well as the 10 mg/kg group which was escalated to 20 mg/kg during the open-label extension (6 months to 1 year). Dyne is initiating registrational cohorts and will update us later this year with their plans for pursuing FDA approval via the accelerated pathway. #exonskipping #CureDuchenne #DMD #FDA #ClinicalTrials https://lnkd.in/g94sfmix

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