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Elexacaftor/tezacaftor/ivacaftor in children aged ≥6 years with cystic fibrosis heterozygous for F508del and a minimal function mutation: results from a 96-week open-label extension study.

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Title: Elexacaftor/tezacaftor/ivacaftor in children aged ≥6 years with cystic fibrosis heterozygous for F508del and a minimal function mutation: results from a 96-week open-label extension study.
Authors: Mall MA; Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité - Universitätsmedizin Berlin, Berlin, Germany.; German Center for Child and Adolescent Health (DZKJ), partner site, Berlin, Germany.; German Center for Lung Research (DZL), associated partner site Berlin, Berlin, Germany.; Co-lead authors., Wainwright CE; Queensland Children's Hospital, University of Queensland, South Brisbane, Australia.; Co-lead authors., Legg J; National Institute for Health Research, Southampton Respiratory Biomedical Research Centre, University Hospitals Southampton NHS Foundation Trust, Southampton, UK.; Southampton Children's Hospital, University Hospitals Southampton NHS Foundation Trust, Southampton, UK., Chilvers M; British Columbia Children's Hospital, University of British Columbia, Vancouver, BC, Canada., Gartner S; Hospital Universitari Vall d'Hebron, Barcelona, Spain., Dittrich AM; Department for Pediatric Pulmonology, Allergology and Neonatology, Hannover Medical School, Hannover, Germany.; BREATH, German Center for Lung Research (DZL), Hannover, Germany., Stehling F; Children's Hospital, University of Duisburg-Essen, Essen, Germany., Conner S; Vertex Pharmaceuticals Incorporated, Boston, MA, USA., Grant S; Vertex Pharmaceuticals Incorporated, Boston, MA, USA., Suresh N; Vertex Pharmaceuticals Incorporated, Boston, MA, USA., Weinstock TG; Vertex Pharmaceuticals Incorporated, Boston, MA, USA., Davies JC; National Heart and Lung Institute, Imperial College London, London, UK j.c.davies@imperial.ac.uk.; Royal Brompton and Harefield Hospitals, Guy's and St Thomas' NHS Trust, London, UK.; European CF Society Lung Clearance Index Core Facility, London, UK.
Corporate Authors: VX20-445-119 Study Group
Source: The European respiratory journal [Eur Respir J] 2025 Jul 14; Vol. 66 (1). Date of Electronic Publication: 2025 Jul 14 (Print Publication: 2025).
Publication Type: Journal Article; Clinical Trial, Phase III; Multicenter Study; Randomized Controlled Trial
Journal Info: Publisher: European Respiratory Society Country of Publication: England NLM ID: 8803460 Publication Model: Electronic-Print Cited Medium: Internet ISSN: 1399-3003 (Electronic) Linking ISSN: 09031936 NLM ISO Abbreviation: Eur Respir J Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Elexacaftor/tezacaftor/ivacaftor in children aged ≥6 years with cystic fibrosis heterozygous for F508del and a minimal function mutation: results from a 96-week open-label extension study.
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  Data: <searchLink fieldCode="AU" term="%22Mall+MA%22">Mall MA</searchLink>; Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité - Universitätsmedizin Berlin, Berlin, Germany.; German Center for Child and Adolescent Health (DZKJ), partner site, Berlin, Germany.; German Center for Lung Research (DZL), associated partner site Berlin, Berlin, Germany.; Co-lead authors.<br /><searchLink fieldCode="AU" term="%22Wainwright+CE%22">Wainwright CE</searchLink>; Queensland Children's Hospital, University of Queensland, South Brisbane, Australia.; Co-lead authors.<br /><searchLink fieldCode="AU" term="%22Legg+J%22">Legg J</searchLink>; National Institute for Health Research, Southampton Respiratory Biomedical Research Centre, University Hospitals Southampton NHS Foundation Trust, Southampton, UK.; Southampton Children's Hospital, University Hospitals Southampton NHS Foundation Trust, Southampton, UK.<br /><searchLink fieldCode="AU" term="%22Chilvers+M%22">Chilvers M</searchLink>; British Columbia Children's Hospital, University of British Columbia, Vancouver, BC, Canada.<br /><searchLink fieldCode="AU" term="%22Gartner+S%22">Gartner S</searchLink>; Hospital Universitari Vall d'Hebron, Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Dittrich+AM%22">Dittrich AM</searchLink>; Department for Pediatric Pulmonology, Allergology and Neonatology, Hannover Medical School, Hannover, Germany.; BREATH, German Center for Lung Research (DZL), Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Stehling+F%22">Stehling F</searchLink>; Children's Hospital, University of Duisburg-Essen, Essen, Germany.<br /><searchLink fieldCode="AU" term="%22Conner+S%22">Conner S</searchLink>; Vertex Pharmaceuticals Incorporated, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Grant+S%22">Grant S</searchLink>; Vertex Pharmaceuticals Incorporated, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Suresh+N%22">Suresh N</searchLink>; Vertex Pharmaceuticals Incorporated, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Weinstock+TG%22">Weinstock TG</searchLink>; Vertex Pharmaceuticals Incorporated, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Davies+JC%22">Davies JC</searchLink>; National Heart and Lung Institute, Imperial College London, London, UK j.c.davies@imperial.ac.uk.; Royal Brompton and Harefield Hospitals, Guy's and St Thomas' NHS Trust, London, UK.; European CF Society Lung Clearance Index Core Facility, London, UK.
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  Data: <searchLink fieldCode="JN" term="%228803460%22">The European respiratory journal</searchLink> [Eur Respir J] 2025 Jul 14; Vol. 66 (1). <i>Date of Electronic Publication: </i>2025 Jul 14 (<i>Print Publication: </i>2025).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22European+Respiratory+Society%22">European Respiratory Society </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>8803460 <i>Publication Model: </i>Electronic-Print <i>Cited Medium: </i>Internet <i>ISSN: </i>1399-3003 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2209031936%22">09031936 </searchLink><i>NLM ISO Abbreviation: </i>Eur Respir J <i>Subsets: </i>MEDLINE
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        Value: 10.1183/13993003.02435-2024
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              Text: 2025 Jul 14
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