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Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis
Author(s) -
Francis X. McCormack,
Yoshikazu Inoue,
Joel Moss,
L.G. Singer,
Charlie Strange,
Koh Nakata,
Alan F. Barker,
Jeffrey T. Chapman,
Mark Brantly,
James Stocks,
Kevin K. Brown,
Joseph P. Lynch,
Hilary J. Goldberg,
Lisa R. Young,
Brent W. Kinder,
Gregory P. Downey,
Eugene J. Sullivan,
Thomas V. Colby,
Roy T. McKay,
Marsha M. Cohen,
Leslie Korbee,
Angelo M. TaveiraDaSilva,
Hye Seung Lee,
Jeffrey P. Krischer,
Bruce C. Trapnell
Publication year - 2011
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1100391
Subject(s) - lymphangioleiomyomatosis , sirolimus , medicine , placebo , lung , urology , vital capacity , discontinuation , gastroenterology , diffusing capacity , lung function , pathology , alternative medicine
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 shown promise in phase 1-2 trials involving patients with LAM.

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