Targeting the Root: Zorevunersen and the Push Toward Disease Modification in Dravet Syndrome

Three epileptologists set the stage on Dravet syndrome, exploring the biological underpinnings of the disease, the cascading burden it places on patients and families, and why this condition demands far more than seizure management.

The panelists discuss the critical shift from seizure-focused management to targeting the underlying genetic etiology of Dravet syndrome, drawing on natural history data that shows developmental impairment progresses independently of seizure control.

The panelists walk through the antisense oligonucleotide mechanism of zorevunersen, explaining how it targets the wild-type SCN1A allele to increase NaV1.1 protein production, and why this approach is uniquely suited to the genetics of Dravet syndrome.

The panelists discuss the four-study development program for zorevunersen -- MONARCH, SWALLOWTAIL, ADMIRAL, and LONGWING -- describing how the clinical research was structured to assess dosing, safety, and both seizure and non-seizure outcomes in an exceptionally treatment-resistant patient population.