Simufilam Regulates Mitochondrial Function in HEK293T TSC2 Knockout Cells

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26 Oct 2026 0 comments

The small molecule simufilam, also called PTI-125, has been reported to reduce cell overgrowth and seizure activity in a mouse model of tuberous sclerosis complex (TSC). TSC is a multisystem developmental disorder caused by mutations in TSC1 or TSC2, resulting in mTOR complex 1 (mTORC1) hyperactivity and downstream cellular changes that contribute to focal malformations of the developing cortex and abnormal growth throughout the body. Here, to better understand the role of simufilam at the...