It is a potent acetylcholinesterase inhibitor, among other properties, and has demonstrated protection against induced seizures in a mouse model of Dravet’s syndrome as well as nerve-agent induced seizures and is being explored as a novel anticonvulsant in a human clinical trial for focal impaired awareness seizures. The gene discussed is ACHE; the disease is encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy.