Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis

X. McCormack, Francis; Inoue, Yoshikazu; Moss, Joel; Singer, L.G.; Strange, Charlie; Nakata, Koh; F. Barker, Alan; T. Chapman, Jeffrey; Brantly, Mark; Stocks, James; K. Brown, Kevin; P. Lynch, Joseph; J. Goldberg, Hilary; R. Young, Lisa; W. Kinder, Brent; P. Downey, Gregory; J. Sullivan, Eugene; V. Colby, Thomas; T. McKay, Roy; M. Cohen, Marsha; Korbee, Leslie; M. Taveira‐DaSilva, Angelo; Seung Lee, Hye; P. Krischer, Jeffrey; C. Trapnell, Bruce

doi:10.1056/nejmoa1100391

Public

Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis

Published in New England Journal of Medicine • Mar 16, 2011

Authors:

Francis X. McCormack

Yoshikazu Inoue

Joel Moss

Abstract

BACKGROUND: Lymphangioleiomyomatosis (LAM) is a progressive, cystic lung disease in women; it is associated with inappropriate activation of mammalian target of rapamycin (mTOR) signaling, which regulates cellular growth and lymphangiogenesis. Sirolimus (also called rapamycin) inhibits mTOR and has ...

Subject

Lymphangioleiomyomatosis

Sirolimus

Medicine

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