Soticlestat, a novel cholesterol 24-hydroxylase inhibitor, reduces seizures and premature death in Dravet syndrome mice.
Nicole A HawkinsManuel JuradoTyler T ThaxtonSamantha E DuarteLevi BarseTetsuya TatsukawaKazuhiro YamakawaToshiya NishiShinichi KondoMaki MiyamotoBrett S AbrahamsMatthew J DuringJennifer A KearneyPublished in: Epilepsia (2021)
This study demonstrates that soticlestat-mediated inhibition of CH24H provides therapeutic benefit for the treatment of Dravet syndrome in mice and has the potential for treatment of DEEs.