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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
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Summary: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 (LCI2.5), along with increases in growth parameters, suggested the potential for early disease modification with LUM/IVA treatment. To further assess the effects of LUM/IVA on CF disease progression in children 2 through 5 years of age using chest magnetic resonance imaging (MRI). ...
Item Description:Gesehen am 29.11.2023
Physical Description:Online Resource
ISSN:2325-6621
DOI:10.1513/AnnalsATS.202208-684OC

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