{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T00:40:55Z","timestamp":1792370455334,"version":"4.4.0"},"reference-count":81,"publisher":"Wiley","issue":"5","content-domain":{"domain":["wiley.com","cochranelibrary.com"],"crossmark-restriction":true},"short-container-title":[],"DOI":"10.1002\/14651858.cd016290","type":"journal-article","created":{"date-parts":[[2026,5,7]],"date-time":"2026-05-07T17:59:37Z","timestamp":1778176777000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["CFTR modulator monotherapy for people with cystic fibrosis with class II\n                    <i>CFTR<\/i>\n                    gene variants (most commonly F508del)"],"prefix":"10.1002","volume":"2026","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-4235-5899","authenticated-orcid":false,"given":"Matthew","family":"Heneghan","sequence":"additional","affiliation":[{"name":"School of Medicine; University of Liverpool; Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4143-5364","authenticated-orcid":false,"given":"Sherie","family":"Smith","sequence":"additional","affiliation":[{"name":"Lifespan and Population Health, School of Medicine; University of Nottingham; Nottingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7836-0151","authenticated-orcid":false,"given":"Nikki","family":"Jahnke","sequence":"additional","affiliation":[{"name":"Lifespan and Population Health, School of Medicine; University of Nottingham; Nottingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9988-2709","authenticated-orcid":false,"given":"Sarah","family":"Nevitt","sequence":"additional","affiliation":[{"name":"Centre for Reviews and Dissemination; University of York; York UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6516-9083","authenticated-orcid":false,"given":"Kevin W","family":"Southern","sequence":"additional","affiliation":[{"name":"Department of Women's and Children's Health; University of Liverpool; Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"supported by the Cochrane Cystic Fibrosis Review Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,5,7]]},"reference":[{"issue":"6","key":"10.1002\/14651858.CD016290-BIB0001|cit1","doi-asserted-by":"crossref","first-page":"575","DOI":"10.1002\/humu.10041","article-title":"Cystic fibrosis: a worldwide analysis of CFTR mutations \u2013 correlation with incidence data and application to screening","volume":"19","author":"Bobadilla","year":"2002","journal-title":"Human Mutation"},{"issue":"4922","key":"10.1002\/14651858.CD016290-BIB0002|cit2","doi-asserted-by":"crossref","first-page":"1066","DOI":"10.1126\/science.2475911","article-title":"Identification of the cystic fibrosis gene \u2013 cloning and characterization of complementary DNA","volume":"245","author":"Riordan","year":"1989","journal-title":"Science"},{"issue":"3","key":"10.1002\/14651858.CD016290-BIB0003|cit3","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1136\/adc.76.3.278","article-title":"Delta F508 in cystic fibrosis: willing but not able","volume":"76","author":"Southern","year":"1997","journal-title":"Archives of Disease in Childhood"},{"issue":"6","key":"10.1002\/14651858.CD016290-BIB0004|cit4","doi-asserted-by":"publisher","first-page":"1480","DOI":"10.1378\/chest.10-2077","article-title":"Cystic fibrosis transmembrane conductance regulator intracellular processing, trafficking, and opportunities for mutation-specific treatment","volume":"139","author":"Rogan","year":"2011","journal-title":"Chest"},{"key":"10.1002\/14651858.CD016290-BIB0005|cit5","unstructured":"Cystic Fibrosis Centre at the Hospital for Sick Children in Toronto Cystic fibrosis mutation database http:\/\/www.genet.sickkids.on.ca\/"},{"issue":"5","key":"10.1002\/14651858.CD016290-BIB0006|cit6","doi-asserted-by":"crossref","first-page":"471","DOI":"10.1046\/j.1469-1809.2003.00028.x","article-title":"The phenotypic consequences of CFTR mutations","volume":"67","author":"Rowntree","year":"2003","journal-title":"Annals of Human Genetics"},{"issue":"3","key":"10.1002\/14651858.CD016290-BIB0007|cit7","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1159\/000102068","article-title":"Cystic fibrosis and formes frustes of CFTR-related disease","volume":"74","author":"Southern","year":"2007","journal-title":"Respiration: International Review of Thoracic Diseases"},{"key":"10.1002\/14651858.CD016290-BIB0008|cit8","unstructured":"2024 Patient Registry Annual Data Report Available at https:\/\/www.cff.org\/medical-professionals\/patient-registry"},{"key":"10.1002\/14651858.CD016290-BIB0009|cit9","unstructured":"2024 Annual Data Report Available at https:\/\/www.cysticfibrosis.org.uk\/about-us\/uk-cf-registry\/reporting-and-resources"},{"issue":"10408","key":"10.1002\/14651858.CD016290-BIB0010|cit10","doi-asserted-by":"publisher","first-page":"1171","DOI":"10.1016\/S0140-6736(23)01609-4","article-title":"CFTR modulator therapy: transforming the landscape of clinical care in cystic fibrosis","volume":"402","author":"Taylor-Cousar","year":"2023","journal-title":"Lancet"},{"issue":"4","key":"10.1002\/14651858.CD016290-BIB0011|cit11","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1038\/ng0895-445","article-title":"Generation and characterization of a delta F508 cystic fibrosis mouse model","volume":"10","author":"Colledge","year":"1995","journal-title":"Nature Genetics"},{"issue":"10","key":"10.1002\/14651858.CD016290-BIB0012|cit12","doi-asserted-by":"crossref","first-page":"2457","DOI":"10.1172\/JCI119788","article-title":"In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR","volume":"100","author":"Rubenstein","year":"1997","journal-title":"Journal of Clinical Investigation"},{"issue":"46","key":"10.1002\/14651858.CD016290-BIB0013|cit13","doi-asserted-by":"publisher","first-page":"18843","DOI":"10.1073\/pnas.1105787108","article-title":"Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809","volume":"108","author":"Van Goor","year":"2011","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"issue":"8","key":"10.1002\/14651858.CD016290-BIB0014|cit14","doi-asserted-by":"publisher","first-page":"653","DOI":"10.1016\/S2213-2600(16)00053-9","article-title":"Newborn screening for cystic fibrosis","volume":"4","author":"Castellani","year":"2016","journal-title":"Lancet Respiratory Medicine"},{"issue":"21","key":"10.1002\/14651858.CD016290-BIB0015|cit15","doi-asserted-by":"publisher","first-page":"1963","DOI":"10.1056\/NEJMoa1301725","article-title":"Risk factors for bronchiectasis in children with cystic fibrosis","volume":"368","author":"Sly","year":"2013","journal-title":"New England Journal of Medicine"},{"issue":"1","key":"10.1002\/14651858.CD016290-BIB0016|cit16","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD009841.pub3","article-title":"Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis","author":"Skilton","year":"2019","journal-title":"Cochrane Database of Systematic Reviews"},{"issue":"11","key":"10.1002\/14651858.CD016290-BIB0017|cit17","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD010966.pub4","article-title":"Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)","author":"Heneghan","journal-title":"Cochrane Database of Systematic Reviews"},{"issue":"8","key":"10.1002\/14651858.CD016290-BIB0018|cit18","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD010966.pub2","article-title":"Correctors (specific therapies for class II CFTR mutations) for cystic fibrosis","author":"Southern","year":"2018","journal-title":"Cochrane Database of Systematic Reviews"},{"issue":"12","key":"10.1002\/14651858.CD016290-BIB0019|cit19","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD010966.pub3","article-title":"Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)","author":"Southern","year":"2020","journal-title":"Cochrane Database of Systematic Reviews"},{"issue":"1","key":"10.1002\/14651858.CD016290-BIB0020|cit20","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1016\/j.jcf.2022.10.002","article-title":"Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis","volume":"22","author":"Southern","year":"2023","journal-title":"Journal of Cystic Fibrosis"},{"key":"10.1002\/14651858.CD016290-BIB0021|cit21","unstructured":"Lumacaftor\u2013ivacaftor for treating cystic fibrosis homozygous for the F508del mutation https:\/\/www.nice.org.uk\/guidance\/ta398"},{"key":"10.1002\/14651858.CD016290-BIB0022|cit22","doi-asserted-by":"crossref","unstructured":"Jahnke N Smith S Southern K Heneghan M Murphy J Nevitt S CFTR modulator monotherapy for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del) www.crd.york.ac.uk\/PROSPERO\/view\/CRD420251090914 2026","DOI":"10.1002\/14651858.CD016290"},{"issue":"6","key":"10.1002\/14651858.CD016290-BIB0023|cit23","doi-asserted-by":"crossref","first-page":"1610","DOI":"10.1378\/chest.08-1190","article-title":"Determination of the minimal clinically important difference scores for the Cystic Fibrosis Questionnaire-Revised respiratory symptom scale in two populations of patients with cystic fibrosis and chronic Pseudomonas aeruginosa airway infection","volume":"135","author":"Quittner","year":"2009","journal-title":"Chest"},{"key":"10.1002\/14651858.CD016290-BIB0024|cit24","unstructured":"Covidence Veritas Health Innovation 2024"},{"key":"10.1002\/14651858.CD016290-BIB0025|cit25","unstructured":"Review Manager (RevMan) The Cochrane Collaboration 2024"},{"key":"10.1002\/14651858.CD016290-BIB0026|cit26","unstructured":"Higgins JP Altman DG Sterne JA Chapter 8: Assessing risk of bias in included studies. In: Higgins JP, Churchill R, Chandler J, Cumpston MS, editor(s), Cochrane Handbook for Systematic Reviews of Interventions Version 5.2.0 (updated June 2017). The Cochrane Collaboration, 2017 Available from https:\/\/www.cochrane.org\/authors\/handbooks-and-manuals\/handbook\/archive\/v5.2.0"},{"key":"10.1002\/14651858.CD016290-BIB0027|cit27","unstructured":"Higgins JP Eldridge S Li T Chapter 23: Including variants on randomized trials. In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA, editor(s). Cochrane Handbook for Systematic Reviews of Interventions Version 6.3 (updated August 2022). Cochrane, 2022 Available from https:\/\/www.cochrane.org\/authors\/handbooks-and-manuals\/handbook\/archive\/v6.3"},{"key":"10.1002\/14651858.CD016290-BIB0028|cit28","unstructured":"Deeks JJ Higgins JP Altman DG Chapter 10: Analysing data and undertaking meta-analyses. In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA, editor(s). Cochrane Handbook for Systematic Reviews of Interventions Version 6.3 (updated August 2022). Cochrane, 2022 Available from https:\/\/www.cochrane.org\/authors\/handbooks-and-manuals\/handbook\/archive\/v6.3"},{"key":"10.1002\/14651858.CD016290-BIB0029|cit29","doi-asserted-by":"crossref","first-page":"2815","DOI":"10.1002\/(SICI)1097-0258(19981230)17:24<2815::AID-SIM110>3.0.CO;2-8","article-title":"Extracting summary statistics to perform meta-analyses of the published literature for survival endpoints","volume":"17","author":"Parmar","year":"1998","journal-title":"Statistics in Medicine"},{"issue":"11","key":"10.1002\/14651858.CD016290-BIB0030|cit30","doi-asserted-by":"crossref","first-page":"3337","DOI":"10.1002\/sim.1303","article-title":"Aggregate data meta-analysis with time-to-event outcomes","volume":"21","author":"Williamson","year":"2002","journal-title":"Statistics in Medicine"},{"issue":"7414","key":"10.1002\/14651858.CD016290-BIB0031|cit31","doi-asserted-by":"crossref","first-page":"557","DOI":"10.1136\/bmj.327.7414.557","article-title":"Measuring inconsistency in meta-analyses","volume":"327","author":"Higgins","year":"2003","journal-title":"BMJ"},{"key":"10.1002\/14651858.CD016290-BIB0032|cit32","unstructured":"Sch\u00fcnemann HJ Higgins JP Vist GE Glasziou P Akl EA Skoetz N Chapter 14: Completing 'Summary of findings' tables and grading the certainty of the evidence (chapter last updated August 2023). In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, et al, editor(s). Cochrane Handbook for Systematic Reviews of Interventions Version 6.5 (updated August 2024). Cochrane, 2024 Available from https:\/\/cochrane.org\/handbook"},{"key":"10.1002\/14651858.CD016290-BIB0033|cit33","unstructured":"Sch\u00fcnemann HJ Vist GE Higgins JP Santesso N Deeks JJ Glasziou P Chapter 15: Interpreting results and drawing conclusions (chapter last updated August 2023). In: Higgins JP, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, et al, editor(s). Cochrane Handbook for Systematic Reviews of Interventions Version 6.5 (updated August 2024). Cochrane, 2024 Available from https:\/\/cochrane.org\/handbook"},{"issue":"5","key":"10.1002\/14651858.CD016290-BIB0034|cit34","doi-asserted-by":"publisher","first-page":"700","DOI":"10.1016\/j.jcf.2019.04.014","article-title":"CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials","volume":"18","author":"Bell","year":"2019","journal-title":"Journal of Cystic Fibrosis"},{"key":"10.1002\/14651858.CD016290-BIB0035|cit35","unstructured":"A study to evaluate multiple doses of GLPG2222 in adult subjects with cystic fibrosis https:\/\/clinicaltrials.gov\/study\/NCT03119649"},{"key":"10.1002\/14651858.CD016290-BIB0036|cit36","first-page":"S42","article-title":"GLPG2222 in subjects with cystic fibrosis homozygous for F508del: results from a phase II study (FLAMINGO)","volume":"17 Suppl 3","author":"Ent","year":"2018","journal-title":"Journal of Cystic Fibrosis"},{"key":"10.1002\/14651858.CD016290-BIB0037|cit37","first-page":"250","article-title":"GLPG2222 in subjects with cystic fibrosis homozygous for F508del: results from a phase II study (FLAMINGO)","volume":"53 Suppl 2","author":"Ent","year":"2018","journal-title":"Pediatric Pulmonology"},{"key":"10.1002\/14651858.CD016290-BIB0038|cit38","doi-asserted-by":"crossref","first-page":"S36","DOI":"10.1016\/S1569-1993(17)30276-X","article-title":"Safety, tolerability and early signs of efficacy with riociguat for the treatment of adult phe508del homozygous cystic fibrosis patients: study design and rationale for the Rio-CF study","volume":"16 Suppl 1","author":"Derichs","year":"2017","journal-title":"Journal of Cystic Fibrosis"},{"key":"10.1002\/14651858.CD016290-BIB0039|cit39","unstructured":"Early signs of efficacy study with riociguat in adult homozygous delta F508 cystic fibrosis patients https:\/\/clinicaltrials.gov\/study\/NCT02170025"},{"key":"10.1002\/14651858.CD016290-BIB0040|cit40","doi-asserted-by":"crossref","first-page":"S67","DOI":"10.1016\/S1569-1993(18)30325-4","article-title":"Riociguat for the treatment of adult phe508del homozygous cystic fibrosis: efficacy data from the phase II Rio-CF study","volume":"17 Suppl 3","author":"Taylor-Cousar","year":"2018","journal-title":"Journal of Cystic Fibrosis"},{"issue":"7","key":"10.1002\/14651858.CD016290-BIB0041|cit41","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1016\/S2213-2600(14)70132-8","article-title":"A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial","volume":"2","author":"Boyle","year":"2014","journal-title":"Lancet. Respiratory Medicine"},{"key":"10.1002\/14651858.CD016290-BIB0042|cit42","unstructured":"A study in people with cystic fibrosis (a rare hereditary pulmonary disease) to assess the efficacy and safety of a combination of two experimental drugs https:\/\/www.clinicaltrialsregister.eu\/ctr-search\/trial\/2012-003990-24\/BE"},{"key":"10.1002\/14651858.CD016290-BIB0043|cit43","unstructured":"Study of VX-809 alone and in combination with VX-770 in cystic fibrosis (CF) patients homozygous or heterozygous for the F508del-CFTR mutation https:\/\/clinicaltrials.gov\/study\/NCT01225211"},{"key":"10.1002\/14651858.CD016290-BIB0044|cit44","first-page":"315","article-title":"The investigational CFTR corrector, VX-809 (lumacaftor) co-administered with the oral potentiator ivacaftor improved CFTR and lung function in F509-8DEL homozygous patients: phase II study results","volume":"47 Suppl 35","author":"Boyle","year":"2012","journal-title":"Pediatric Pulmonology"},{"key":"10.1002\/14651858.CD016290-BIB0045|cit45","doi-asserted-by":"crossref","first-page":"S14","DOI":"10.1016\/S1569-1993(13)60043-0","article-title":"Lumacaftor, an investigational CFTR corrector, in combination with ivacaftor, a CFTR potentiator, in CF patients with the F508del-CFTR mutation: phase 2 interim analysis","volume":"12 Suppl 1","author":"Boyle","year":"2013","journal-title":"Journal of Cystic Fibrosis"},{"issue":"2","key":"10.1002\/14651858.CD016290-BIB0046|cit46","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1513\/AnnalsATS.201609-689OC","article-title":"Lumacaftor\/ivacaftor treatment of patients with cystic fibrosis heterozygous for F508del-CFTR","volume":"14","author":"Rowe","year":"2017","journal-title":"Annals of the American Thoracic Society"},{"key":"10.1002\/14651858.CD016290-BIB0047|cit47","first-page":"287","article-title":"VX-809, an investigational CFTR corrector, in combination with VX-770, an investigational CFTR potentiator, in subjects with CF and homozygous for the F508DEL-CFTR mutation","volume":"46 Suppl 34","author":"Boyle","year":"2011","journal-title":"Pediatric Pulmonology"},{"issue":"7","key":"10.1002\/14651858.CD016290-BIB0048|cit48","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1016\/S2213-2600(14)70132-8","article-title":"Supplementary Appendix to \"A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: A phase 2 randomised controlled trial\"","volume":"2","author":"Boyle","year":"2014","journal-title":"Lancet. Respiratory Medicine"},{"issue":"2","key":"10.1002\/14651858.CD016290-BIB0049|cit49","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1513\/AnnalsATS.201609-689OC","article-title":"Lumacaftor\/ivacaftor treatment of patients with cystic fibrosis heterozygous for F508del-CFTR","volume":"14","author":"Rowe","year":"2017","journal-title":"Annals of the American Thoracic Society"},{"issue":"2","key":"10.1002\/14651858.CD016290-BIB0050|cit50","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1513\/AnnalsATS.201609-689OC","article-title":"Lumacaftor\/ivacaftor treatment of patients with cystic fibrosis heterozygous for F508del-CFTR","volume":"14","author":"Rowe","year":"2017","journal-title":"Annals of the American Thoracic Society"},{"issue":"1","key":"10.1002\/14651858.CD016290-BIB0051|cit51","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1136\/thoraxjnl-2011-200393","article-title":"Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation","volume":"67","author":"Clancy","year":"2012","journal-title":"Thorax"},{"key":"10.1002\/14651858.CD016290-BIB0052|cit52","unstructured":"Study of VX-809 in cystic fibrosis subjects with the \u2206F508-CFTR gene mutation https:\/\/clinicaltrials.gov\/study\/NCT00865904"},{"key":"10.1002\/14651858.CD016290-BIB0053|cit53","doi-asserted-by":"crossref","first-page":"S20","DOI":"10.1016\/S1569-1993(10)60074-4","article-title":"Clinical evaluation of VX-809, a novel investigational oral F508del-CFTR corrector, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation","volume":"9 Suppl 1","author":"Clancy","year":"2010","journal-title":"Journal of Cystic Fibrosis"},{"issue":"S34","key":"10.1002\/14651858.CD016290-BIB0054|cit54","first-page":"283","article-title":"Variability of nasal potential difference measurements in clinical testing of CFTR modulators","volume":"46 Suppl 34","author":"Clancy","year":"2011","journal-title":"Pediatric Pulmonology"},{"issue":"S33","key":"10.1002\/14651858.CD016290-BIB0055|cit55","first-page":"298","article-title":"A phase II, randomized, placebo-controlled, clinical trial of four doses of VX-809 in CF patients homozygous for the F508del CFTR mutation","volume":"45 Suppl 33","author":"Clancy","year":"2010","journal-title":"Pediatric Pulmonology"},{"key":"10.1002\/14651858.CD016290-BIB0056|cit56","first-page":"308","article-title":"Safety, tolerability, and pharmacokinetics of the intravenous S-nitrosoglutathione reductase inhibitor N6022: an ascending-dose study in subjects homozygous for the F508DEL-CFTR mutation","volume":"49 Suppl 38","author":"Donaldson","year":"2014","journal-title":"Pediatric Pulmonology"},{"key":"10.1002\/14651858.CD016290-BIB0057|cit57","first-page":"154","article-title":"Novel modifiers of CFTR: emerging clinical experience with GSNOR inhibitors","volume":"49 Suppl 38","author":"Donaldson","year":"2014","journal-title":"Pediatric Pulmonology"},{"key":"10.1002\/14651858.CD016290-BIB0058|cit58","unstructured":"Safety and pharmacokinetic study of N6022 in subjects with cystic fibrosis homozygous for the F508del CFTR mutation (SNO1) https:\/\/clinicaltrials.gov\/study\/NCT01746784"},{"issue":"3","key":"10.1002\/14651858.CD016290-BIB0059|cit59","doi-asserted-by":"crossref","first-page":"371","DOI":"10.1016\/j.jcf.2017.01.009","article-title":"Pharmacokinetics and safety of cavosonstat (N91115) in healthy and cystic fibrosis adults homozygous for F508DEL-CFTR","volume":"16","author":"Donaldson","year":"2017","journal-title":"Journal of Cystic Fibrosis"},{"key":"10.1002\/14651858.CD016290-BIB0060|cit60","unstructured":"Study of N91115 in patients with cystic fibrosis homozygous F508del-CFTR mutation (SNO4) https:\/\/clinicaltrials.gov\/study\/NCT02275936"},{"key":"10.1002\/14651858.CD016290-BIB0061|cit61","first-page":"293","article-title":"Safety and pharmacokinetics of N91115 in patients with cystic fibrosis homozygous for the F508DEL-CFTR mutation","volume":"50 Suppl 41","author":"Donaldson","year":"2015","journal-title":"Pediatric Pulmonology"},{"issue":"3","key":"10.1002\/14651858.CD016290-BIB0062|cit62","doi-asserted-by":"crossref","first-page":"371","DOI":"10.1016\/j.jcf.2017.01.009","article-title":"Pharmacokinetics and safety of cavosonstat (N91115) in healthy and cystic fibrosis adults homozygous for F508DEL-CFTR","volume":"16","author":"Donaldson","year":"2017","journal-title":"Journal of Cystic Fibrosis"},{"issue":"Suppl 3","key":"10.1002\/14651858.CD016290-BIB0063|cit63","doi-asserted-by":"crossref","first-page":"S42","DOI":"10.1016\/S1569-1993(18)30251-0","article-title":"Safety, pharmacokinetics and pharmacodynamics of the CFTR corrector FDL169","volume":"17","author":"Horsley","year":"2018","journal-title":"Journal of Cystic Fibrosis"},{"key":"10.1002\/14651858.CD016290-BIB0064|cit64","unstructured":"A study to evaluate safety, PK and PD of FDL169 in cystic fibrosis subjects https:\/\/clinicaltrials.gov\/study\/NCT03093714"},{"key":"10.1002\/14651858.CD016290-BIB0065|cit65","first-page":"252","article-title":"Novel CFTR corrector FDL169: safety, pharmacokinetics and pharmacodynamics","volume":"53 (S2)","author":"Horsley","year":"2018","journal-title":"Pediatric Pulmonology"},{"key":"10.1002\/14651858.CD016290-BIB0066|cit66","doi-asserted-by":"crossref","first-page":"S42","DOI":"10.1016\/S1569-1993(18)30251-0","article-title":"Novel CFTR corrector FDL169: safety, pharmacokinetics and pharmacodynamics","volume":"17 Suppl 3","author":"Horsley","year":"2018","journal-title":"Journal of Cystic Fibrosis"},{"issue":"2","key":"10.1002\/14651858.CD016290-BIB0067|cit67","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1002\/ppul.10041","article-title":"A phase I randomized, multicenter trial of CPX in adult subjects with mild cystic fibrosis","volume":"33","author":"McCarty","year":"2002","journal-title":"Pediatric Pulmonology"},{"key":"10.1002\/14651858.CD016290-BIB0068|cit68","first-page":"276","article-title":"Safety of a phase I double-blind placebo-controlled dose escalation trial of oral CPX in adult CF patients","volume":"26 Suppl 17","author":"Aitken","year":"1998","journal-title":"Pediatric Pulmonology"},{"issue":"2","key":"10.1002\/14651858.CD016290-BIB0069|cit69","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1002\/ppul.10043","article-title":"Use of nasal potential difference and sweat chloride as outcome measures in multicenter clinical trials in subjects with cystic fibrosis","volume":"33","author":"Ahrens","year":"2002","journal-title":"Pediatric Pulmonology"},{"key":"10.1002\/14651858.CD016290-BIB0070|cit70","first-page":"276","article-title":"Multicenter phase I trial of CPX in adults patients with mild CF: results of nasal potential difference measurements","volume":"26 Suppl 17","author":"McCarty","year":"1998","journal-title":"Pediatric Pulmonology"},{"issue":"2","key":"10.1002\/14651858.CD016290-BIB0071|cit71","doi-asserted-by":"crossref","first-page":"484","DOI":"10.1164\/ajrccm.157.2.9706088","article-title":"A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function","volume":"157","author":"Rubenstein","year":"1998","journal-title":"American Journal of Respiratory and Critical Care Medicine"},{"key":"10.1002\/14651858.CD016290-BIB0072|cit72","first-page":"272","article-title":"A randomized, double blind, placebo-controlled trial of sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function","volume":"Suppl 14","author":"Rubenstein","year":"1997","journal-title":"Pediatric Pulmonology"},{"issue":"1","key":"10.1002\/14651858.CD016290-BIB0073|cit73","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1006\/mthe.2002.0639","article-title":"Evidence of CFTR function in cystic fibrosis after systemic administration of 4-phenylbutyrate","volume":"6","author":"Zeitlin","year":"2002","journal-title":"Molecular Therapy"},{"issue":"3","key":"10.1002\/14651858.CD016290-BIB0074|cit74","doi-asserted-by":"publisher","first-page":"375","DOI":"10.1016\/j.jcf.2024.05.007","article-title":"Standards for the care of people with cystic fibrosis (CF); Planning for a longer life","volume":"23","author":"Gramegna","year":"2024","journal-title":"Journal of Cystic Fibrosis"},{"key":"10.1002\/14651858.CD016290-BIB0075|cit75","doi-asserted-by":"publisher","first-page":"456","DOI":"10.1016\/j.jcf.2022.01.009","article-title":"Worldwide rates of diagnosis and effective treatment for cystic fibrosis","volume":"21","author":"Guo","year":"2022","journal-title":"Journal of Cystic Fibrosis"},{"issue":"6","key":"10.1002\/14651858.CD016290-BIB0076|cit76","doi-asserted-by":"publisher","first-page":"1622","DOI":"10.1002\/ppul.26954","article-title":"International disparities in diagnosis and treatment access for cystic fibrosis","volume":"59","author":"Guo","year":"2024","journal-title":"Pediatric Pulmonology"},{"key":"10.1002\/14651858.CD016290-BIB0077|cit77","doi-asserted-by":"publisher","first-page":"1163391","DOI":"10.3389\/fphar.2023.1163391","article-title":"Real-world disparities and ethical considerations with access to CFTR modulator drugs: mind the gap!","volume":"14","author":"Zampoli","year":"2023","journal-title":"Frontiers in Pharmacology"},{"issue":"1","key":"10.1002\/14651858.CD016290-BIB0078|cit78","doi-asserted-by":"publisher","first-page":"690","DOI":"10.4081\/mrm.2020.690","article-title":"Novel therapeutic approaches for the management of cystic fibrosis","volume":"15","author":"Jaques","year":"2020","journal-title":"Multidisciplinary Respiratory Medicine"},{"key":"10.1002\/14651858.CD016290-BIB0079|cit79","doi-asserted-by":"publisher","first-page":"4818","DOI":"10.1136\/bmj.d4818","article-title":"Health inequalities and cystic fibrosis","volume":"343","author":"Taylor-Robinson","year":"2011","journal-title":"BMJ"},{"issue":"1","key":"10.1002\/14651858.CD016290-BIB0080|cit80","doi-asserted-by":"publisher","first-page":"693","DOI":"10.1038\/s41467-023-36244-2","article-title":"Future therapies for cystic fibrosis","volume":"14","author":"Allen","year":"2023","journal-title":"Nature Communications"},{"key":"10.1002\/14651858.CD016290-BIB0081|cit81","doi-asserted-by":"crossref","first-page":"840","DOI":"10.1136\/thorax-2023-220100","article-title":"The top 10 research priorities in cystic fibrosis developed by a partnership between people with CF and healthcare providers","volume":"78","author":"Rowbotham","year":"2023","journal-title":"Thorax"}],"container-title":["Cochrane Database of Systematic Reviews"],"original-title":[],"language":"en","deposited":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T00:04:50Z","timestamp":1792368290000},"score":1,"resource":{"primary":{"URL":"http:\/\/doi.wiley.com\/10.1002\/14651858.CD016290"}},"subtitle":[],"editor":[{"name":"Cochrane Central Editorial Service","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2026,5,7]]},"references-count":81,"journal-issue":{"issue":"5","published-online":{"date-parts":[[2026,5]]}},"URL":"https:\/\/doi.org\/10.1002\/14651858.cd016290","relation":{},"ISSN":["1465-1858"],"issn-type":[{"value":"1465-1858","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,5,7]]}}}