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Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis.
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Abstract
Angiomyolipomas in patients with the tuberous sclerosis complex or sporadic lymphangioleiomyomatosis are associated with mutations in tuberous sclerosis genes resulting in constitutive activation of the mammalian target of rapamycin (mTOR). The drug sirolimus suppresses mTOR signaling.