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.

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Title: 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.
Authors: Stahl M; Department of Pediatric Respiratory Medicine, Immunology, and Critical Care Medicine and.; German Center for Lung Research, Associated Partner Site, Berlin, Germany.; Berlin Institute of Health at Charité - Universitätsmedizin Berlin, Berlin, Germany., Roehmel J; Department of Pediatric Respiratory Medicine, Immunology, and Critical Care Medicine and., Eichinger M; Translational Lung Research Center Heidelberg, German Center for Lung Research, Heidelberg, Germany.; Department of Diagnostic and Interventional Radiology.; Department of Diagnostic and Interventional Radiology with Nuclear Medicine, Thoraxklinik, and., Doellinger F; Department of Radiology, Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany., Naehrlich L; Department of Pediatrics, Justus Liebig University Giessen, Giessen, Germany.; Universities of Giessen and Marburg Lung Center, German Center for Lung Research, Giessen, Germany., Kopp MV; Pediatric Respiratory Medicine, Department of Pediatrics, Inselspital, University of Bern, Bern, Switzerland.; Airway Research Center North, German Center for Lung Research, Grosshansdorf, Germany., Dittrich AM; Department for Pediatric Pulmonology, Allergology, and Neonatology and.; BREATH, German Center for Lung Research, Hannover Medical School, Hannover, Germany; and., Lee C; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts., Sommerburg O; Translational Lung Research Center Heidelberg, German Center for Lung Research, Heidelberg, Germany.; Division of Pediatric Pulmonology and Allergy and Cystic Fibrosis Center, Department of Pediatrics, Heidelberg University Hospital, Heidelberg, Germany., Tian S; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts., Xu T; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts., Wu P; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts., Joshi A; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts., Ray P; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts., Duncan ME; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts., Wielpütz MO; Translational Lung Research Center Heidelberg, German Center for Lung Research, Heidelberg, Germany.; Department of Diagnostic and Interventional Radiology.; Department of Diagnostic and Interventional Radiology with Nuclear Medicine, Thoraxklinik, and., Mall MA; Department of Pediatric Respiratory Medicine, Immunology, and Critical Care Medicine and.; German Center for Lung Research, Associated Partner Site, Berlin, Germany.; Berlin Institute of Health at Charité - Universitätsmedizin Berlin, Berlin, Germany.
Source: Annals of the American Thoracic Society [Ann Am Thorac Soc] 2023 Aug; Vol. 20 (8), pp. 1144-1155.
Publication Type: Randomized Controlled Trial; Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: American Thoracic Society Country of Publication: United States NLM ID: 101600811 Publication Model: Print Cited Medium: Internet ISSN: 2325-6621 (Electronic) Linking ISSN: 23256621 NLM ISO Abbreviation: Ann Am Thorac Soc Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2325-6621
DOI:10.1513/AnnalsATS.202208-684OC