Researchers at University College London and Great Ormond Street Hospital reported clinical-trial results showing the investigational gene-regulation therapy zorevunersen produced marked, sustained reductions in seizure frequency for children with Dravet syndrome. Trial data published in the New England Journal of Medicine and accompanying reports indicate treated patients experienced 59–91% fewer seizures over follow-up and improvements in daily functioning. Dravet syndrome is most often caused by loss-of-function mutations in SCN1A; zorevunersen is designed to address that genetic deficit directly. A randomized phase 3 study is ongoing to test efficacy in a larger, controlled cohort; regulators and investors will watch those results for potential registration implications.