As an example tuberous sclerosis complex (TSC), an autosomal dominant genetic disorder, is characterized by mutations in TSC1 or TSC2 resulting in an inappropriate mTORC1 signaling which is thought to be responsible for the manifestation of this condition, like renal angiomyolipomas (AMLs) and subependymal giant astrocytoma (SEGA); mTOR inhibitor everolimus has been used for the treatment of both adult and children with AMLs and SEGA according to the data from the clinical trials EXIST-1 and EXIST-2. Here, TSC1 is linked to subependymal giant cell astrocytoma.