mTOR inhibitors were reported by 31 patients with TSC (everolimus, n = 30; sirolimus n = 1), primarily to treat multiple organ manifestations, including 21 patients using mTOR inhibitors for angiomyolipoma, 17 patients for epilepsy, 15 patients for subependymal giant cell astrocytoma, and 6 patients for skin manifestation (multiple treatment targets possible). The gene discussed is MTOR; the disease is subependymal giant cell astrocytoma.