Clinical trials led by UCL and Great Ormond Street Hospital reported that zorevunersen, a gene‑regulatory therapy for Dravet syndrome, produced dramatic seizure reductions—between ~59% and 91% across dosing cohorts—and functional improvements in pediatric patients. Results published in the New England Journal of Medicine show robust seizure control and quality‑of‑life gains across an 81‑patient cohort and support expedited late‑stage development. Investigators emphasized favorable safety and tolerability across doses, while sponsors and advocacy groups called the outcomes 'unprecedented' for this drug‑resistant childhood epilepsy. A randomized Phase 3 is underway; regulators and payers will now evaluate long‑term durability, age ranges, and potential for accelerated pathways given high unmet need and mortality risk in Dravet syndrome.