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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.

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          Author and article information

          Journal
          N Engl J Med
          The New England journal of medicine
          Massachusetts Medical Society
          1533-4406
          0028-4793
          Jan 10 2008
          : 358
          : 2
          Affiliations
          [1 ] Division of Nephrology and Hypertension, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229-3039, USA. john.bissler@cchmc.org
          Article
          358/2/140 NIHMS116421
          10.1056/NEJMoa063564
          3398441
          18184959
          6be74cf0-7f45-4ace-bfcc-3a87fdd76dd8
          2008 Massachusetts Medical Society
          History

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