A large number of randomized controlled clinical trials with mTOR inhibitors have shown efficacy in the treatment of several clinical organ manifestations of TSC, most importantly in patients with subependymal giant cell astrocytoma (SEGA), seizures (treatment resistant with focal onset), and angiomyolipoma (AML), as well as cutaneous manifestations [9,10,11,12]. The gene discussed is MTOR; the disease is tuberous sclerosis.