Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis by Bissler John J, McCormack Francis X, Young Lisa R, Elwing Jean M, Chuck Gail, Leonard Jennifer M, Schmithorst Vincent J, Laor Tal, Brody Alan S, Bean Judy, Salisbury Shelia, Franz David N in The New England journal of medicine (2008). PubMed

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.

[ hide abstract ]

Discussed In Paper