Efficacy and safety of low-dose Sirolimus in Lymphangioleiomyomatosis

Efficacy and safety of low-dose Sirolimus in Lymphangioleiomyomatosis

Abstract Background Lymphangioleiomyomatosis is a rare disease caused by unregulated activation of mammalian target of rapamycin (mTOR) signalling pathway. Sirolimus showed efficacy in a phase 3 trial of patients with lymphangioleiomyomatosis, but the optimal dose remains unclear. Methods We investi...

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Bibliographic Details
Main Authors: Hee-Young Yoon, Jung Jin Hwang, Dong Soon Kim, Jin Woo Song
Format: Article
Language:English
Published: BMC 2018-11-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Lymphangioleiomyomatosis
Sirolimus
Low dose
Respiratory function tests
Treatment outcome
Online Access:http://link.springer.com/article/10.1186/s13023-018-0946-8

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