STXBP1 and encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy: For example, scn1lab−/− mutants (a model of Dravet syndrome) have served as a screening platform and have pinpointed clemizole as a potential drug candidate for Dravet syndrome patients (Baraban et al., 2013), and more recently also for STXBP1 mutations carriers (Moog and Baraban, 2022).