Also known as: Nav1.1, NaV1.1
Gene editing repaired a Dravet syndrome mutation in mice, a prenatal hemophilia A therapy showed maternal safety in sheep, and researchers advanced toward a genetic test for valproic acid use in pregnancy.
New treatments for rare pediatric neurological conditions show promise, with zorevunersen reducing seizures in Dravet syndrome patients and an SMA therapy under FDA review following stem cell research breakthroughs.
Stoke Therapeutics presented long-term Dravet syndrome data showing up to 80% seizure reductions sustained through three years, outlined its Phase 3 EMPEROR trial design targeting mid-2027 data, and disclosed first patient dosing in its ADOA program.
