Stoke Therapeutics is currently conducting a phase 1/2a study of STK-001, an 2MOE PS ASO designed to skip a nonproductive exon in SCN1A gene, in Dravet syndrome (Table 2; Stoke, 2020). Here, SCN1A is linked to encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy.