First, clinical data from patients with SCN1A‐related Dravet syndrome seem to confirm a relevant efficacy of the antisense oligonucleotide zorevunersen (former STK001),158, 159 which mediates a targeted augmentation of nuclear gene output with increased production of the productive SCN1A and functional messenger RNA (mRNA) by modulation of a splicing event. The gene discussed is SCN1A; the disease is encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy.