Wave life sciences announces positive interim data from forward-53 clinical trial evaluating wve-n531 in boys with duchenne muscular dystrophy amenable to exon 53 skipping

Mean muscle content-adjusted dystrophin expression of 9.0% and unadjusted dystrophin of 5.5%, with high consistency across participants, in a prespecified analysis; dystrophin was comprised of two isoforms consistent with becker muscular dystrophy patients who display milder disease

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