Abstract
In The Lancet, John Bissler and colleagues1 report the first randomised, double-blind, placebo-controlled, phase 3 trial of everolimus, an inhibitor of mammalian target of rapamycin (mTOR), in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis, and angiomyolipoma. Their findings suggest a clear advantage for everolimus over placebo in reducing angiomyolipoma volume with an acceptable safety profile in patients with tuberous sclerosis.
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