piperidines has been researched along with Sudden-Unexpected-Death-in-Epilepsy* in 1 studies
1 other study(ies) available for piperidines and Sudden-Unexpected-Death-in-Epilepsy
Article | Year |
---|---|
Soticlestat, a novel cholesterol 24-hydroxylase inhibitor, reduces seizures and premature death in Dravet syndrome mice.
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. In this study, the novel compound soticlestat, a CH24H inhibitor, was administered to Scn1a. Soticlestat treatment reduced seizure burden, protected against hyperthermia-induced seizures, and completely prevented SUDEP in Scn1a. 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. Topics: Animals; Cholesterol 24-Hydroxylase; Epilepsies, Myoclonic; Epilepsy; Epileptic Syndromes; Mice; Mortality, Premature; Mutation; NAV1.1 Voltage-Gated Sodium Channel; Piperidines; Pyridines; Seizures; Seizures, Febrile; Sudden Unexpected Death in Epilepsy | 2021 |