A gene regulation approach for Dravet syndrome, zorevunersen, produced dramatic seizure reductions and functional gains in pediatric patients in early clinical testing, according to data published in the New England Journal of Medicine. Trial cohorts experienced 59–91% reductions in seizure frequency and reported improved day‑to‑day functioning. The therapy targets the dominant SCN1A mutation underlying most Dravet cases and was delivered across a range of doses to children aged two to 18. Side effects were mostly moderate or mild; a Phase III randomized trial is underway to confirm efficacy and safety. These results mark a rare example of clinically meaningful disease modification in a severe pediatric epileptic encephalopathy and accelerate the regulatory and commercial conversations around one‑time gene‑targeted interventions in neurodevelopmental disease.