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Evaluating Long-Term Effectiveness of Cystic Fibrosis Modulator Therapies after Rapid Adoption: A Dual-Approach Study.

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Title: Evaluating Long-Term Effectiveness of Cystic Fibrosis Modulator Therapies after Rapid Adoption: A Dual-Approach Study.
Authors: Miranda Afonso P; Department of Biostatistics and.; Department of Epidemiology, Erasmus Medical Center, Rotterdam, the Netherlands., Zhou GC; Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, Tennessee.; Division of Biostatistics and Epidemiology and., Su W; Division of Biostatistics and Epidemiology and.; Eli Lilly and Company, Indianapolis, Indiana., Burgel PR; Institut Cochin, Inserm U1016, Universite Paris Cité, Paris, France.; Respiratory Medicine and Cystic Fibrosis National Reference Center, Cochin Hospital, Paris, France.; ERN-Lung CF Network, Frankfurt, Germany., Cromwell E; Cystic Fibrosis Foundation, Bethesda, Maryland., Goss CH; Department of Medicine and.; Department of Pediatrics, School of Medicine, and., Keogh RH; Department of Medical Statistics, London School of Hygiene and Tropical Medicine, London, United Kingdom., Liou TG; Division of Respiratory, Critical Care, and Occupational Pulmonary Medicine, Department of Internal Medicine, School of Medicine, and.; Center for Quantitative Biology, University of Utah, Salt Lake City, Utah., Marshall BC; Cystic Fibrosis Foundation, Bethesda, Maryland., Mayer-Hamblett N; Department of Pediatrics, School of Medicine, and.; Department of Biostatistics, University of Washington, Seattle, Washington.; Cystic Fibrosis Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington., Morgan WJ; Department of Pediatrics, University of Arizona, Tucson, Arizona., Ostrenga JS; Cystic Fibrosis Foundation, Bethesda, Maryland., Pasta DJ; DMA Statistics Inc., Palo Alto, California., Schechter MS; Division of Pulmonary Medicine, Department of Pediatrics, Children's Hospital of Richmond, Virginia Commonwealth University, Richmond, Virginia., Stanojevic S; Department of Community Health and Epidemiology, Dalhousie University, Halifax, Nova Scotia, Canada., Wainwright CE; Children's Health Queensland Hospital and Health Service, Brisbane, Queensland, Australia.; Child Health Research Centre, The University of Queensland, South Brisbane, Queensland, Australia., Szczesniak RD; Division of Biostatistics and Epidemiology and.; Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.; Department of Pediatrics, University of Cincinnati, Cincinnati, Ohio; and., Andrinopoulou ER; Department of Biostatistics and.; Department of Epidemiology, Erasmus Medical Center, Rotterdam, the Netherlands.; Statistics and Data Science Innovation Hub, GSK, Amsterdam, the Netherlands.
Source: Annals of the American Thoracic Society [Ann Am Thorac Soc] 2026 Jan 01; Vol. 23 (1), pp. 89-99.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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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  Data: <searchLink fieldCode="AU" term="%22Miranda+Afonso+P%22">Miranda Afonso P</searchLink>; Department of Biostatistics and.; Department of Epidemiology, Erasmus Medical Center, Rotterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Zhou+GC%22">Zhou GC</searchLink>; Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, Tennessee.; Division of Biostatistics and Epidemiology and.<br /><searchLink fieldCode="AU" term="%22Su+W%22">Su W</searchLink>; Division of Biostatistics and Epidemiology and.; Eli Lilly and Company, Indianapolis, Indiana.<br /><searchLink fieldCode="AU" term="%22Burgel+PR%22">Burgel PR</searchLink>; Institut Cochin, Inserm U1016, Universite Paris Cité, Paris, France.; Respiratory Medicine and Cystic Fibrosis National Reference Center, Cochin Hospital, Paris, France.; ERN-Lung CF Network, Frankfurt, Germany.<br /><searchLink fieldCode="AU" term="%22Cromwell+E%22">Cromwell E</searchLink>; Cystic Fibrosis Foundation, Bethesda, Maryland.<br /><searchLink fieldCode="AU" term="%22Goss+CH%22">Goss CH</searchLink>; Department of Medicine and.; Department of Pediatrics, School of Medicine, and.<br /><searchLink fieldCode="AU" term="%22Keogh+RH%22">Keogh RH</searchLink>; Department of Medical Statistics, London School of Hygiene and Tropical Medicine, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Liou+TG%22">Liou TG</searchLink>; Division of Respiratory, Critical Care, and Occupational Pulmonary Medicine, Department of Internal Medicine, School of Medicine, and.; Center for Quantitative Biology, University of Utah, Salt Lake City, Utah.<br /><searchLink fieldCode="AU" term="%22Marshall+BC%22">Marshall BC</searchLink>; Cystic Fibrosis Foundation, Bethesda, Maryland.<br /><searchLink fieldCode="AU" term="%22Mayer-Hamblett+N%22">Mayer-Hamblett N</searchLink>; Department of Pediatrics, School of Medicine, and.; Department of Biostatistics, University of Washington, Seattle, Washington.; Cystic Fibrosis Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington.<br /><searchLink fieldCode="AU" term="%22Morgan+WJ%22">Morgan WJ</searchLink>; Department of Pediatrics, University of Arizona, Tucson, Arizona.<br /><searchLink fieldCode="AU" term="%22Ostrenga+JS%22">Ostrenga JS</searchLink>; Cystic Fibrosis Foundation, Bethesda, Maryland.<br /><searchLink fieldCode="AU" term="%22Pasta+DJ%22">Pasta DJ</searchLink>; DMA Statistics Inc., Palo Alto, California.<br /><searchLink fieldCode="AU" term="%22Schechter+MS%22">Schechter MS</searchLink>; Division of Pulmonary Medicine, Department of Pediatrics, Children's Hospital of Richmond, Virginia Commonwealth University, Richmond, Virginia.<br /><searchLink fieldCode="AU" term="%22Stanojevic+S%22">Stanojevic S</searchLink>; Department of Community Health and Epidemiology, Dalhousie University, Halifax, Nova Scotia, Canada.<br /><searchLink fieldCode="AU" term="%22Wainwright+CE%22">Wainwright CE</searchLink>; Children's Health Queensland Hospital and Health Service, Brisbane, Queensland, Australia.; Child Health Research Centre, The University of Queensland, South Brisbane, Queensland, Australia.<br /><searchLink fieldCode="AU" term="%22Szczesniak+RD%22">Szczesniak RD</searchLink>; Division of Biostatistics and Epidemiology and.; Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.; Department of Pediatrics, University of Cincinnati, Cincinnati, Ohio; and.<br /><searchLink fieldCode="AU" term="%22Andrinopoulou+ER%22">Andrinopoulou ER</searchLink>; Department of Biostatistics and.; Department of Epidemiology, Erasmus Medical Center, Rotterdam, the Netherlands.; Statistics and Data Science Innovation Hub, GSK, Amsterdam, the Netherlands.
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  Data: <searchLink fieldCode="JN" term="%22101600811%22">Annals of the American Thoracic Society</searchLink> [Ann Am Thorac Soc] 2026 Jan 01; Vol. 23 (1), pp. 89-99.
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