Dravet syndrome is a rare genetic disease caused by a mutation of the SCN1A gene, which has not yet had an approved disease-modifying drug[2]. Researchers are now testing two new approaches—antisense oligonucleotide therapy and viral vector therapy—that should target the genetic defect directly instead of just suppressing seizures[1]. Both therapies work by increasing the production of healthy SCN1A protein from the intact copy of the gene, which would compensate for the insufficient production caused by the mutation[1]. Early clinical trials showed encouraging results with significant reductions in seizures and improvements in non-seizure areas such as speech development and communication skills[1]. One of these therapies recently entered phase three clinical trials, an important step toward regulatory approval[1]. Although these discoveries are exciting, wider availability of these drugs is not expected for several years[1]. When available, they are likely to complement existing drugs rather than completely replace them[1].