Article
Soticlestat, a novel cholesterol 24-hydroxylase inhibitor, reduces seizures and premature death in Dravet syndrome mice.
Epilepsia - 1 Nov 2021
Hawkins Nicole A, Jurado Manuel, Thaxton Tyler T, Duarte Samantha E, Barse Levi, Tatsukawa Tetsuya, Yamakawa Kazuhiro, Nishi Toshiya, Kondo Shinichi, Miyamoto Maki, Abrahams Brett S, During Matthew J, Kearney Jennifer A
Abstract excerpt
OBJECTIVE: Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) most often caused by de novo pathogenic variants in SCN1A. Individuals with Dravet syndrome rarely achieve seizure control and have significantly elevated risk for sudden unexplained death in epilepsy (SUDEP). Heterozygous deletion of Scn1a in mice (Scn1a+/- ) recapitulates several core phenotypes, including...
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