Emerging Disease-Modifying Therapies in Dravet Syndrome

Panelists discuss how promising disease-modifying therapies including antisense oligonucleotide and viral vector treatments are currently in clinical trials and target the underlying SCN1A gene defect, rather than just treating seizure symptoms, showing encouraging results in seizure reduction and nonseizure outcomes such as language development, though these therapies will likely complement rather than replace existing medications when they become available in several years.