Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for F508del-CFTR: A Phase 2, Open-Label Clinical Trial.
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| Title: | Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for F508del-CFTR: A Phase 2, Open-Label Clinical Trial. |
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| Authors: | Stahl M; Department of Pediatric Respiratory Medicine, Immunology, and Critical Care Medicine and.; German Center for Lung Research (DZL), Associated Partner Site, Berlin, Germany.; Berlin Institute of Health at Charité-Universitätsmedizin, Berlin, Germany., Roehmel J; Department of Pediatric Respiratory Medicine, Immunology, and Critical Care Medicine and.; German Center for Lung Research (DZL), Associated Partner Site, Berlin, Germany.; Berlin Institute of Health at Charité-Universitätsmedizin, Berlin, Germany., Eichinger M; Translational Lung Research Center Heidelberg (TLRC), German Center for Lung Research (DZL), Heidelberg, Germany.; Department of Diagnostic and Interventional Radiology and.; Department of Diagnostic and Interventional Radiology with Nuclear Medicine, Thoraxklinik at University Hospital Heidelberg, Heidelberg, Germany., 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 (UGMLC), German Center for Lung Research (DZL), Giessen, Germany., Kopp MV; Pediatric Respiratory Medicine, Department of Pediatrics, Inselspital, University of Bern, Bern, Switzerland.; Airway Research Center North (ARCN), German Center for Lung Research (DZL), Grosshansdorf, Germany., Dittrich AM; Department for Pediatric Pulmonology, Allergology, and Neonatology, and.; BREATH, German Center for Lung Research (DZL), Hannover Medical School, Hannover, Germany., Sommerburg O; Translational Lung Research Center Heidelberg (TLRC), German Center for Lung Research (DZL), Heidelberg, Germany.; Division of Pediatric Pulmonology and Allergy and Cystic Fibrosis Center, Department of Pediatrics, Heidelberg University Hospital, Heidelberg, Germany., Ray P; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts; and., Maniktala A; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts; and.; ICON plc, Global Strategic Solutions, Raleigh, North Carolina., Xu T; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts; and., Conner S; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts; and., Joshi A; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts; and., Mascia M; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts; and., Wielpütz MO; Translational Lung Research Center Heidelberg (TLRC), German Center for Lung Research (DZL), Heidelberg, Germany.; Department of Diagnostic and Interventional Radiology and.; Department of Diagnostic and Interventional Radiology with Nuclear Medicine, Thoraxklinik at University Hospital Heidelberg, Heidelberg, Germany., Mall MA; Department of Pediatric Respiratory Medicine, Immunology, and Critical Care Medicine and.; German Center for Lung Research (DZL), Associated Partner Site, Berlin, Germany.; Berlin Institute of Health at Charité-Universitätsmedizin, Berlin, Germany. |
| Source: | Annals of the American Thoracic Society [Ann Am Thorac Soc] 2024 Nov; Vol. 21 (11), pp. 1550-1559. |
| Publication Type: | Journal Article; Randomized Controlled Trial; Clinical Trial, Phase II; Multicenter Study |
| Journal Info: | Publisher: American Thoracic Society Country of Publication: England 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 |
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| ISSN: | 2325-6621 |
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| DOI: | 10.1513/AnnalsATS.202402-201OC |
