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Effects of lumacaftor/ivacaftor on cystic fibrosis disease progression in children 2 through 5 years of age homozygous for F508del-CFTR: a phase 2 placebo-controlled clinical trial

Lumacaftor/ivacaftor (LUM/IVA) was shown to be safe and well tolerated in children 2 through 5 years of age with cystic fibrosis (CF) homozygous for F508del-CFTR in a Phase 3 open-label study. Improvements in sweat chloride concentration, markers of pancreatic function, and lung clearance index2.5 (...

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Main Authors: Stahl, Mirjam (Author) , Roehmel, Jobst (Author) , Eichinger, Monika (Author) , Doellinger, Felix (Author) , Naehrlich, Lutz (Author) , Kopp, Matthias V. (Author) , Dittrich, Anna-Maria (Author) , Lee, Christopher (Author) , Sommerburg, Olaf (Author) , Tian, Simon (Author) , Xu, Tu (Author) , Wu, Pan (Author) , Joshi, Aniket (Author) , Ray, Partha (Author) , Duncan, Margaret E. (Author) , Wielpütz, Mark Oliver (Author) , Mall, Marcus A. (Author)
Format: Article (Journal)
Language:English
Published: Aug 2023
In: Annals of the American Thoracic Society
Year: 2023, Volume: 20, Issue: 8, Pages: 1144-1155
ISSN:2325-6621
DOI:10.1513/AnnalsATS.202208-684OC
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1513/AnnalsATS.202208-684OC
Verlag, kostenfrei, Volltext: https://www.atsjournals.org/doi/10.1513/AnnalsATS.202208-684OC
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Author Notes:Mirjam Stahl, Jobst Roehmel, Monika Eichinger, Felix Doellinger, Lutz Naehrlich, Matthias V. Kopp, Anna-Maria Dittrich, Christopher Lee, Olaf Sommerburg, Simon Tian, Tu Xu, Pan Wu, Aniket Joshi, Partha Ray, Margaret E. Duncan, Mark O. Wielpütz, and Marcus A. Mall

MARC

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