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Maximising opportunity for therapeutic success: sequential participation in cystic fibrosis nucleic acid-based therapy trials.

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Title: Maximising opportunity for therapeutic success: sequential participation in cystic fibrosis nucleic acid-based therapy trials.
Authors: Mayer-Hamblett N; Seattle Children's Research Institute, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA; Department of Biostatistics, University of Washington, Seattle, WA, USA. Electronic address: nicole.hamblett@seattlechildrens.org., Kerper NR; Cystic Fibrosis Foundation, Bethesda, MD, USA., Clancy JP; Cystic Fibrosis Foundation, Bethesda, MD, USA., Davies JC; National Heart and Lung Institute, Imperial College London, London, UK; Royal Brompton Hospital, Guy's and St Thomas' NHS Foundation Trust, London, UK., Taylor-Cousar JL; Departments of Internal Medicine and Pediatrics, National Jewish Health, Denver, CO, USA., Donaldson SH; Division of Pulmonary and Critical Care Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA., Bell SC; Department of Respiratory Medicine, Gold Coast University Hospital, Southport, QLD, Australia; School of Medicine and Dentistry, Health Group, Griffith University, Southport, QLD, Australia., Jain R; Pulmonary and Critical Care Medicine, University of Texas Southwestern Medical Center, Dallas, TX, USA., Simmonds NJ; National Heart and Lung Institute, Imperial College London, London, UK; Adult Cystic Fibrosis Centre, Royal Brompton Hospital, Guy's and St Thomas' NHS Foundation Trust, London, UK., Mall MA; Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany; German Centre for Lung Research (DZL), Berlin, Germany; German Center for Child and Adolescent Health (DZKJ), Berlin, Germany., Goss CH; Seattle Children's Research Institute, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA; Division of Pulmonary, Critical Care and Sleep Medicine, Department of Medicine, University of Washington, Seattle, WA, USA., Rayment JH; Division of Respiratory Medicine, BC Children's Hospital, Vancouver, BC, Canada; Department of Pediatrics, University of British Columbia, Vancouver, BC, Canada., Setiawan L; Seattle Children's Research Institute, Seattle, WA, USA., Pilewski JM; Division of Pulmonary, Allergy, Critical Care and Sleep Medicine, Department of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
Source: The Lancet. Respiratory medicine [Lancet Respir Med] 2025 Dec; Vol. 13 (12), pp. 1108-1118. Date of Electronic Publication: 2025 Jul 30.
Publication Type: Journal Article; Review
Journal Info: Publisher: Elsevier Country of Publication: England NLM ID: 101605555 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2213-2619 (Electronic) Linking ISSN: 22132600 NLM ISO Abbreviation: Lancet Respir Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Maximising opportunity for therapeutic success: sequential participation in cystic fibrosis nucleic acid-based therapy trials.
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  Data: <searchLink fieldCode="AU" term="%22Mayer-Hamblett+N%22">Mayer-Hamblett N</searchLink>; Seattle Children's Research Institute, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA; Department of Biostatistics, University of Washington, Seattle, WA, USA. Electronic address: nicole.hamblett@seattlechildrens.org.<br /><searchLink fieldCode="AU" term="%22Kerper+NR%22">Kerper NR</searchLink>; Cystic Fibrosis Foundation, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Clancy+JP%22">Clancy JP</searchLink>; Cystic Fibrosis Foundation, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Davies+JC%22">Davies JC</searchLink>; National Heart and Lung Institute, Imperial College London, London, UK; Royal Brompton Hospital, Guy's and St Thomas' NHS Foundation Trust, London, UK.<br /><searchLink fieldCode="AU" term="%22Taylor-Cousar+JL%22">Taylor-Cousar JL</searchLink>; Departments of Internal Medicine and Pediatrics, National Jewish Health, Denver, CO, USA.<br /><searchLink fieldCode="AU" term="%22Donaldson+SH%22">Donaldson SH</searchLink>; Division of Pulmonary and Critical Care Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.<br /><searchLink fieldCode="AU" term="%22Bell+SC%22">Bell SC</searchLink>; Department of Respiratory Medicine, Gold Coast University Hospital, Southport, QLD, Australia; School of Medicine and Dentistry, Health Group, Griffith University, Southport, QLD, Australia.<br /><searchLink fieldCode="AU" term="%22Jain+R%22">Jain R</searchLink>; Pulmonary and Critical Care Medicine, University of Texas Southwestern Medical Center, Dallas, TX, USA.<br /><searchLink fieldCode="AU" term="%22Simmonds+NJ%22">Simmonds NJ</searchLink>; National Heart and Lung Institute, Imperial College London, London, UK; Adult Cystic Fibrosis Centre, Royal Brompton Hospital, Guy's and St Thomas' NHS Foundation Trust, London, UK.<br /><searchLink fieldCode="AU" term="%22Mall+MA%22">Mall MA</searchLink>; Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany; German Centre for Lung Research (DZL), Berlin, Germany; German Center for Child and Adolescent Health (DZKJ), Berlin, Germany.<br /><searchLink fieldCode="AU" term="%22Goss+CH%22">Goss CH</searchLink>; Seattle Children's Research Institute, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA; Division of Pulmonary, Critical Care and Sleep Medicine, Department of Medicine, University of Washington, Seattle, WA, USA.<br /><searchLink fieldCode="AU" term="%22Rayment+JH%22">Rayment JH</searchLink>; Division of Respiratory Medicine, BC Children's Hospital, Vancouver, BC, Canada; Department of Pediatrics, University of British Columbia, Vancouver, BC, Canada.<br /><searchLink fieldCode="AU" term="%22Setiawan+L%22">Setiawan L</searchLink>; Seattle Children's Research Institute, Seattle, WA, USA.<br /><searchLink fieldCode="AU" term="%22Pilewski+JM%22">Pilewski JM</searchLink>; Division of Pulmonary, Allergy, Critical Care and Sleep Medicine, Department of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
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  Data: <searchLink fieldCode="JN" term="%22101605555%22">The Lancet. Respiratory medicine</searchLink> [Lancet Respir Med] 2025 Dec; Vol. 13 (12), pp. 1108-1118. <i>Date of Electronic Publication: </i>2025 Jul 30.
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              Text: 2025 Dec
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