Genetic pathogenesis of the epileptogenic lesions in Tuberous Sclerosis Complex: Therapeutic targeting of the mTOR pathway

•Overactivation of the mTOR pathway is responsible for TSC-related epilepsy.•Mammalian target of rapamycin (mTOR) inhibitors offer a targeted therapeutic and systemic option in TSC.•In mouse models, rapamycin prevented epilepsy and reverted histological abnormalities.•Everolimus is effective in different seizure types with an acceptable safety profile.•Everolimus is more effective in younger children.