Wave Life Sciences Shares 48-Week Data from FORWARD-53 Trial of WVE-N531 in Duchenne Muscular Dystrophy

Dear Members of the Duchenne community,We are excited to share positive data following 48 weeks of dosing in the FORWARD-53 clinical trial evaluating the investigational molecule WVE-N531 in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.FORWARD-53 is a Phase 1b/2a trial of 11 boys aged 5-11 (10 ambulatory and 1 non-ambulatory), which is designed to evaluate safety, dystrophin expression, muscle health and functional outcomes. The latest data after 48 weeks of 10 mg/kg dosing every other week include muscle biopsies for eight of 11 boys (all ambulatory) while safety and functional out come assessments were available for all participants.