Perfect match: mTOR inhibitors and tuberous sclerosis complex

Highlights Hyperactivation of mammalian target of rapamycin (mTOR) is essential in the pathogenesis of tuberous sclerosis complex (TSC) and can serve as a therapeutic target. mTOR inhibitors have shown considerable success in multiple clinical trials for the treatment of TSC, including neurological, pulmonary, cardiac, renal, and cutaneous phenotypes. mTOR inhibitors are associated with adverse events, which should be considered during the management of TSC. Indicators to predict mTOR inhibitor efficacy are required to select patients who are likely to benefit from such therapy.