{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,10]],"date-time":"2026-03-10T22:19:36Z","timestamp":1773181176192,"version":"3.50.1"},"reference-count":45,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"content-domain":{"domain":["clinicalkey.fr","em-consulte.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Archives de P\u00e9diatrie"],"published-print":{"date-parts":[[2023,1]]},"DOI":"10.1016\/j.arcped.2022.11.019","type":"journal-article","created":{"date-parts":[[2022,12,9]],"date-time":"2022-12-09T15:19:32Z","timestamp":1670599172000},"page":"1-9","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":4,"title":["CFTR pharmacological modulators: A great advance in cystic fibrosis management"],"prefix":"10.1016","volume":"30","author":[{"given":"P.","family":"Foucaud","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7842-3378","authenticated-orcid":false,"given":"J.C.","family":"Mercier","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.arcped.2022.11.019_bib0001","doi-asserted-by":"crossref","first-page":"2195","DOI":"10.1016\/S0140-6736(20)32542-3","article-title":"Cystic fibrosis","volume":"397","author":"Shteinberg","year":"2021","journal-title":"Lancet"},{"key":"10.1016\/j.arcped.2022.11.019_bib0002","unstructured":"Registre fran\u00e7ais de la mucoviscidose: bilan des donn\u00e9es 2020. [Internet]. https:\/\/www.registredelamuco.org (accessed 7 September 2022)."},{"key":"10.1016\/j.arcped.2022.11.019_bib0003","doi-asserted-by":"crossref","first-page":"1059","DOI":"10.1126\/science.2772657","article-title":"Identification of the cystic fibrosis gene: chromosome walking and jumping","volume":"245","author":"Rommens","year":"1989","journal-title":"Science"},{"key":"10.1016\/j.arcped.2022.11.019_bib0004","doi-asserted-by":"crossref","first-page":"1066","DOI":"10.1126\/science.2475911","article-title":"Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA","volume":"245","author":"Riordan","year":"1989","journal-title":"Science"},{"key":"10.1016\/j.arcped.2022.11.019_bib0005","doi-asserted-by":"crossref","first-page":"1066","DOI":"10.1126\/science.2570460","article-title":"Identification of the cystic fibrosis gene: genetic analysis","volume":"245","author":"Kerem","year":"1989","journal-title":"Science"},{"key":"10.1016\/j.arcped.2022.11.019_bib0006","doi-asserted-by":"crossref","first-page":"2233","DOI":"10.1056\/NEJMra0910061","article-title":"Airway mucus function and dysfunction","volume":"363","author":"Fahy","year":"2010","journal-title":"N Engl J Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0007","doi-asserted-by":"crossref","first-page":"158","DOI":"10.1016\/S2213-2600(12)70057-7","article-title":"A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect","volume":"1","author":"Boyle","year":"2013","journal-title":"Lancet Respir Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0008","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1164\/rccm.201701-0134OC","article-title":"Airway mucosal defense is key to genomic regulation of cystic fibrosis lung disease severity","volume":"197","author":"Polineli","year":"2018","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0009","doi-asserted-by":"crossref","first-page":"S5","DOI":"10.1016\/j.jcf.2019.11.003","article-title":"The genetics and genomics of cystic fibrosis","volume":"19","author":"Sharma","year":"2020","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.arcped.2022.11.019_bib0010","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1056\/NEJMra1300109","article-title":"Origins of cystic fibrosis lung disease","volume":"372","author":"Stoltz","year":"2015","journal-title":"N Engl J Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0011","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1007\/s00018-016-2391-y","article-title":"Role of CFTR in epithelial physiology","volume":"74","author":"Saint-Cricq","year":"2017","journal-title":"Cell Mol Life Sci"},{"key":"10.1016\/j.arcped.2022.11.019_bib0012","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1016\/j.chest.2018.04.036","article-title":"Ion channel modulators in cystic fibrosis","volume":"54","author":"Gentzch","year":"2018","journal-title":"Chest"},{"key":"10.1016\/j.arcped.2022.11.019_bib0013","doi-asserted-by":"crossref","first-page":"1251","DOI":"10.1016\/0092-8674(93)90353-R","article-title":"Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis","volume":"73","author":"Welsh","year":"1993","journal-title":"Cell"},{"key":"10.1016\/j.arcped.2022.11.019_bib0014","doi-asserted-by":"crossref","first-page":"8382","DOI":"10.1038\/ncomms9382","article-title":"Genome-wide association meta-analysis identifies five modifier loci of lung disease severity in cystic fibrosis","volume":"6","author":"Corvol","year":"2015","journal-title":"Nat Commun"},{"key":"10.1016\/j.arcped.2022.11.019_bib0015","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1038\/347358a0","article-title":"Expression of cystic fibrosis transmembrane regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells","volume":"347","author":"Rich","year":"1990","journal-title":"Nature"},{"key":"10.1016\/j.arcped.2022.11.019_bib0016","doi-asserted-by":"crossref","first-page":"483","DOI":"10.1016\/j.cell.2017.06.041","article-title":"Conformational changes of CFTR upon phosphorylation and ATP binding","volume":"170","author":"Zhang","year":"2017","journal-title":"Cell"},{"key":"10.1016\/j.arcped.2022.11.019_bib0017","doi-asserted-by":"crossref","first-page":"37235","DOI":"10.1074\/jbc.M205932200","article-title":"High-affinity activators of cystic fibrosis transmembrane conductance regulator (CFTR) chloride conductance identified by high-throughput screening","volume":"277","author":"Ma","year":"2002","journal-title":"J Biol Chem"},{"key":"10.1016\/j.arcped.2022.11.019_bib0018","doi-asserted-by":"crossref","DOI":"10.1016\/j.jpeds.2016.10.045","article-title":"Differences in outcomes between early and late diagnosis of cystic fibrosis in the newborn screening era","volume":"181","author":"Coffey","year":"2017","journal-title":"J Pediatr"},{"key":"10.1016\/j.arcped.2022.11.019_bib0019","first-page":"163","article-title":"[Newborn screening for cystic fibrosis in France: practical aspects and perspectives] d\u00e9pistage n\u00e9onatal de la mucoviscidose en France: aspects pratiques et perspectives","volume":"2","author":"Munck","year":"2019","journal-title":"Perfect P\u00e9diatr"},{"key":"10.1016\/j.arcped.2022.11.019_bib0020","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1016\/j.jcf.2014.08.007","article-title":"on behalf of the CHOPIN study group. Effectiveness of newborn screening for cystic fibrosis determined with real-life data","volume":"14","author":"Van der Ploeg","year":"2015","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.arcped.2022.11.019_bib0021","doi-asserted-by":"crossref","first-page":"1663","DOI":"10.1056\/NEJMoa1105185","article-title":"A CFTR potentiator in patients with cystic fibrosis and the G551D mutation","volume":"365","author":"Ramsey","year":"2011","journal-title":"N Engl J Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0022","doi-asserted-by":"crossref","first-page":"2347","DOI":"10.1378\/chest.128.4.2347","article-title":"Development and validation of the cystic fibrosis questionnaire in the United States: a health-related quality-of-life measure for cystic fibrosis","volume":"128","author":"Quittner","year":"2005","journal-title":"Chest"},{"key":"10.1016\/j.arcped.2022.11.019_bib0023","doi-asserted-by":"crossref","first-page":"1219","DOI":"10.1164\/rccm.201301-0153OC","article-title":"On behalf of the VX08-770-103 (ENVISION) study group. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation","volume":"187","author":"Davies","year":"2013","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0024","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1016\/S2213-2600(15)00545-7","article-title":"On behalf of the KIWI study group. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study","volume":"4","author":"Davies","year":"2016","journal-title":"Lancet Respir Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0025","doi-asserted-by":"crossref","first-page":"545","DOI":"10.1016\/S2213-2600(18)30202-9","article-title":"ARRIVAL study group. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase-3 single-arm study","volume":"6","author":"Rosenfeld","year":"2018","journal-title":"Lancet Respir Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0026","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1164\/rccm.202008-3177OC","article-title":"On behalf of the ARRIVAL study group. Ivacaftor in infants aged 4 to <12 months with cystic fibrosis and a gating mutation. Results of a two-part phase 3 clinical trial","volume":"203","author":"Davies","year":"2020","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0027","doi-asserted-by":"crossref","first-page":"220","DOI":"10.1056\/NEJMoa1409547","article-title":"For the TRAFFIC and TRANSPORT study groups. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR","volume":"373","author":"Wainwright","year":"2015","journal-title":"N Engl J Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0028","doi-asserted-by":"crossref","first-page":"912","DOI":"10.1164\/rccm.201608-1754OC","article-title":"On behalf of the VX13-809-11 Part B investigator group. Lumacaftor\/ivacaftor in patients aged 6-11 years with cystic fibrosis and homogeneous for F508del-CFTR","volume":"195","author":"Milla","year":"2017","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0029","doi-asserted-by":"crossref","first-page":"977","DOI":"10.1016\/S2213-2600(21)00069-2","article-title":"Long-term safety of lumacaftor-ivacaftor in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label extension trial","volume":"9","author":"Hoppe","year":"2021","journal-title":"Lancet Respir Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0030","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1164\/rccm.201906-1227OC","article-title":"For the French cystic fibrosis reference network study group. Real-life safety and effectiveness of lumacaftor-ivacaftor in patients with cystic fibrosis","volume":"201","author":"Burgel","year":"2020","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0031","doi-asserted-by":"crossref","first-page":"2013","DOI":"10.1056\/NEJMoa1709846","article-title":"Tezacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del","volume":"377","author":"Taylor-Cousar","year":"2017","journal-title":"N Engl J Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0032","doi-asserted-by":"crossref","first-page":"2024","DOI":"10.1056\/NEJMoa1709847","article-title":"Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis","volume":"377","author":"Rowe","year":"2017","journal-title":"N Engl J Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0033","doi-asserted-by":"crossref","first-page":"708","DOI":"10.1016\/j.jcf.2019.06.009","article-title":"On behalf of the VX15-661-113 investigator group. A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis","volume":"18","author":"Walker","year":"2019","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.arcped.2022.11.019_bib0034","doi-asserted-by":"crossref","first-page":"1612","DOI":"10.1056\/NEJMoa1807120","article-title":"For the VX16-445-001 study group. VX-445-tezacaftor-ivacaftor in patients with cystic fibrosis and one or two alleles Phe508del alleles","volume":"379","author":"Keating","year":"2018","journal-title":"N Engl J Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0035","doi-asserted-by":"crossref","first-page":"1809","DOI":"10.1056\/NEJMoa1908639","article-title":"For the VX17-445-102 group. Elexacaftor-Tezacaftor-Ivacaftor for cystic fibrosis with a single Phe508del allele","volume":"381","author":"Middleton","year":"2019","journal-title":"N Engl J Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0036","doi-asserted-by":"crossref","first-page":"1940","DOI":"10.1016\/S0140-6736(19)32597-8","article-title":"On behalf of the VX-17-445-103 trial group. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial","volume":"394","author":"Heijerman","year":"2019","journal-title":"Lancet"},{"key":"10.1016\/j.arcped.2022.11.019_bib0037","doi-asserted-by":"crossref","first-page":"815","DOI":"10.1056\/NEJMoa2100665","article-title":"For the VX18-445-104 study group. Triple therapy for cystic fibrosis Phe508del-gating and Phe508del-residual function genotypes","volume":"385","author":"Barry","year":"2021","journal-title":"N Engl J Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0038","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1016\/S2213-2600(21)00454-9","article-title":"On behalf of the VX18-445-109 study group. Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial","volume":"10","author":"Sutharsan","year":"2022","journal-title":"Lancet Respir Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0039","doi-asserted-by":"crossref","first-page":"1522","DOI":"10.1164\/rccm.202102-0509OC","article-title":"For the VX18-445-106 study group. A phase 3 open-label study of elexacaftor\/tezacaftor\/ivacaftor in children 6 through 11 years of age with cystic fibrosis and at least one F508del allele","volume":"203","author":"Zemanick","year":"2021","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0040","doi-asserted-by":"crossref","first-page":"977","DOI":"10.1164\/rccm.202006-2433OC","article-title":"Lung clearance index to track acute respiratory events in school-age children with cystic fibrosis","volume":"203","author":"Perrem","year":"2021","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0041","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1164\/rccm.202011-4153OC","article-title":"For the French cystic fibrosis reference network study group. Rapid improvement after starting elexacaftor-tezacaftor-ivacaftor in patients with cystic fibrosis and advanced pulmonary disease","volume":"204","author":"Burgel","year":"2021","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0042","doi-asserted-by":"crossref","first-page":"584","DOI":"10.1164\/rccm.202109-2121LE","article-title":"Major decrease in lung transplantation for patients with cystic fibrosis in France","volume":"205","author":"Martin","year":"2022","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0043","doi-asserted-by":"crossref","first-page":"1116","DOI":"10.1164\/rccm.201901-0145OC","article-title":"Correlating cystic fibrosis transmembrane conductance regulator function with clinical features to inform precision treatment of cystic fibrosis","volume":"199","author":"McCague","year":"2019","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.arcped.2022.11.019_bib0044","doi-asserted-by":"crossref","first-page":"546","DOI":"10.3390\/genes11050546","article-title":"Transcriptomic and proteostasis networks of CFTR and the development of small molecule modulators for the treatment of cystic fibrosis lung disease","volume":"11","author":"Strub","year":"2020","journal-title":"Genes"},{"key":"10.1016\/j.arcped.2022.11.019_bib0045","doi-asserted-by":"crossref","first-page":"604","DOI":"10.1165\/rcmb.2019-0291OC","article-title":"Novel correctors and potentiators enhance translational readthrough in CFTR nonsense mutations","volume":"64","author":"Mutyam","year":"2021","journal-title":"Am J Respir Cell Mol Biol"}],"container-title":["Archives de P\u00e9diatrie"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0929693X22002469?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0929693X22002469?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,15]],"date-time":"2025-10-15T14:55:21Z","timestamp":1760540121000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0929693X22002469"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,1]]},"references-count":45,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2023,1]]}},"alternative-id":["S0929693X22002469"],"URL":"https:\/\/doi.org\/10.1016\/j.arcped.2022.11.019","relation":{},"ISSN":["0929-693X"],"issn-type":[{"value":"0929-693X","type":"print"}],"subject":[],"published":{"date-parts":[[2023,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"CFTR pharmacological modulators: A great advance in cystic fibrosis management","name":"articletitle","label":"Article Title"},{"value":"Archives de P\u00e9diatrie","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.arcped.2022.11.019","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 Published by Elsevier Masson SAS on behalf of French Society of Pediatrics.","name":"copyright","label":"Copyright"}]}}