MTOR inhibitors received clinical approval as oral therapy for specific TSC lesions, such as everolimus to treat subependymal giant cell astrocytoma,8 renal angiomyolipoma,9 and as adjunctive treatment of TSC-partial onset seizures10; and sirolimus (rapamycin) for lymphangioleiomyomatosis.11 The gene discussed is MTOR; the disease is lymphangioleiomyomatosis.