Everolimus for the treatment of lymphangioleiomyomatosis: a phase II study

J. Goldberg, Hilary; Harari, Sergio; Cottin, Vincent; O. Rosas, Iván; Peters, Elizabeth; Biswal, Shibadas; Cheng, Yi; Khindri, Sanjeev; M. Kovarik, John; Ma, Shenglin; X. McCormack, Francis; P. Henske, Elizabeth

doi:10.1183/09031936.00210714

Public

Everolimus for the treatment of lymphangioleiomyomatosis: a phase II study

Published in European Respiratory Journal • Jun 25, 2015

Authors:

Hilary J. Goldberg

Sergio Harari

Vincent Cottin

Abstract

Lymphangioleiomyomatosis is a rare, progressive cystic lung disorder characterised by dysregulated activation of mammalian target of rapamycin (mTOR) signalling.This was a phase IIa, multicentre, open-label study of the mTOR inhibitor everolimus (2.5 mg·day(-1) escalated to 10 mg·day(-1)) in 24 wome...

Subject

Lymphangioleiomyomatosis

Everolimus

Medicine

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