Article
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.
Annals of the American Thoracic Society - 1 Aug 2023
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
Abstract excerpt
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 index2.5 (LCI2.5), along with increases in growth parameters, suggested the potential for early disease...
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