Dravet syndrome therapeutics: where are we, what works, and what’s next?

DS therapeutics are converging on disease modification: gene-regulatory approaches lead, and targeted small molecules extend, mechanism-based care. We outline a practical precision ecosystem with four pillars: (i) early genetic screening with genotype-informed prediction, (ii) pharmacogenomic-guided prescribing, (iii) patient-specific variant modeling to clarify mechanism and optimize drug selection, and (iv) targeted restorative gene-modifying therapies that address causation. Together, these elements shift DS care toward individualized, mechanism-matched treatment providing seizure control as well as measurable gains in communication, learning, mobility, sleep, and autonomic stability.