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In the majority of cases, TSC results from mutations in the <jats:italic>TSC1<\/jats:italic> and <jats:italic>TSC2<\/jats:italic> genes, leading to the overactivation of the mammalian target of rapamycin (mTOR) signalling pathway, which controls several cell functions, including cell growth, proliferation, and survival. The establishment of a connection between TSC and mTOR led to the clinical use of drugs known as mTOR inhibitors (like rapamycin, also known as sirolimus and everolimus), which are becoming an increasingly interesting tool in the management of TSC\u2010associated features, such as subependymal giant cell astrocytomas, renal angiomyolipomas, and also epilepsy. However, the intrinsic characteristics of these drugs and their systemic effects in such a heterogeneous condition pose many challenges in clinical practice, so that some questions remain unanswered. This article provides an overview of the pharmacological aspects of mTOR inhibitors about the clinical trials leading to their approval in TSC\u2010related conditions and exposes current challenges and future directions associated with this promising therapeutic line.<\/jats:p>","DOI":"10.1155\/2017\/9820181","type":"journal-article","created":{"date-parts":[[2017,3,12]],"date-time":"2017-03-12T21:02:03Z","timestamp":1489352523000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":53,"title":["Recent Advances and Challenges of mTOR Inhibitors Use in the Treatment of Patients with Tuberous Sclerosis 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