An experimental gene-regulation therapy, zorevunersen, produced large reductions in seizure frequency for children with Dravet syndrome in multicenter studies led by University College London and Great Ormond Street Hospital, data published in the New England Journal of Medicine show. Across dose cohorts, investigators reported between 59% and 91% fewer seizures versus baseline in treated patients and improvements in daily functioning and quality of life. The trials enrolled pediatric patients aged roughly 2–18 and focused on restoring function at the disease-causing SCN1A locus; zorevunersen is designed to address the underlying genetic defect rather than only suppress seizures. A randomized phase 3 study is ongoing to confirm these open-label and dose-ranging safety and efficacy signals before regulatory filing plans are finalized.