type of treatment gene strategy/treatment Reference
Aetiology-based preventive treatments and in progress treatments Preventive teraphy TSC1/TSC2 Vigabatrin prior to sz onset Jóźwiak et al 2011
Pyridoxin-dependent epilepsy Maternal administration Pyridoxin Stockler et al 2010
Clinical ongoing trial SCN1A ASO/STK-001 https://dravetsyndromenews.com/; the MONARCH trial (NCT04442295) Stoke Therapeutics
nonsense SCN1A mutations Ataluren https://clinicaltrials.gov/ct2/show/NCT02758626
CDKL5 deficiency Ataluren https://clinicaltrials.gov/ct2/show/NCT02758626
In progress (tested only in animal models) SCN8A ASO Lenk et al 2020
SCN1A ASO Lenk et al 2020
EPM2A/EPM2B Gys1-ASO Ahonen et al 2021