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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

RATIONALE: 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...

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Detalles Bibliográficos
Autores principales:	Stahl, Mirjam, Roehmel, Jobst, Eichinger, Monika, Doellinger, Felix, Naehrlich, Lutz, Kopp, Matthias V., Dittrich, Anna-Maria, Lee, Christopher, Sommerburg, Olaf, Tian, Simon, Xu, Tu, Wu, Pan, Joshi, Aniket, Ray, Partha, Duncan, Margaret E., Wielpütz, Mark O., Mall, Marcus A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Thoracic Society 2023
Materias:	Original Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10405608/ https://www.ncbi.nlm.nih.gov/pubmed/36943405 http://dx.doi.org/10.1513/AnnalsATS.202208-684OC

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