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Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare CFTR variants: a viewpoint.
Burgel PR, Sermet-Gaudelus I, Girodon E, Kanaan R, Le Bihan J, Remus N, Ravoninjatovo B, Grenet D, Porzio M, Houdouin V, Le Clainche-Viala L, Durieu I, Nove-Josserand R, Languepin J, Coltey B, Guillaumot A, Audousset C, Chiron R, Weiss L, Fajac I, Da Silva J, Martin C; French CF Reference Network study group. Burgel PR, et al. Among authors: weiss l. Eur Respir J. 2024 Jan 25;63(1):2301959. doi: 10.1183/13993003.01959-2023. Print 2024 Jan. Eur Respir J. 2024. PMID: 38242629 No abstract available.
[French guidelines for sweat test practice and interpretation for cystic fibrosis neonatal screening].
Sermet-Gaudelus I, Munck A, Rota M, Roussey M, Feldmann D, Nguyen-Khoa T; Groupe de travail "Dépistage néonatal" de la Fédération des centres de ressources et de compétences de la mucoviscidose. Sermet-Gaudelus I, et al. Arch Pediatr. 2010 Sep;17(9):1349-58. doi: 10.1016/j.arcped.2010.06.021. Epub 2010 Aug 16. Arch Pediatr. 2010. PMID: 20719482 French.
Reference percentiles for FEV(1) and BMI in European children and adults with cystic fibrosis.
Boëlle PY, Viviani L, Busson PF, Olesen HV, Ravilly S, Stern M, Assael BM, Barreto C, Drevinek P, Thomas M, Krivec U, Mei-Zahav M, Vibert JF, Clement A, Mehta A, Corvol H; French CF Modifier Gene Study Investigators; European CF Registry Working Group. Boëlle PY, et al. Orphanet J Rare Dis. 2012 Sep 7;7:64. doi: 10.1186/1750-1172-7-64. Orphanet J Rare Dis. 2012. PMID: 22958330 Free PMC article.
Central venous thrombosis and thrombophilia in cystic fibrosis: A prospective study.
Munck A, Kheniche A, Alberti C, Hubert D, Martine RG, Nove-Josserand R, Pin I, Bremont F, Chiron R, Couderc L, Dalphin ML, Darviot E, Delaisi B, Dominique S, Durieu I, Fanton A, Fayon M, Gérardin M, Giniès JL, Giraut C, Grenet D, Guillot M, Huet F, Le Bourgeois M, Murris-Epin M, Ramel S, Sardet A, Sermet-Gaudelus I, Varaigne F, Wanin S, Weiss L, Hurtaud MF. Munck A, et al. Among authors: weiss l. J Cyst Fibros. 2015 Jan;14(1):97-103. doi: 10.1016/j.jcf.2014.05.015. Epub 2014 Aug 5. J Cyst Fibros. 2015. PMID: 25107684 Free article.
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, Ratjen F, Rowe SM, Waltz D, Boyle MP; TRAFFIC Study Group; TRANSPORT Study Group. Wainwright CE, et al. N Engl J Med. 2015 Jul 16;373(3):220-31. doi: 10.1056/NEJMoa1409547. Epub 2015 May 17. N Engl J Med. 2015. PMID: 25981758 Free PMC article. Clinical Trial.
[Management of infants whose diagnosis is inconclusive at neonatal screening for cystic fibrosis].
Sermet-Gaudelus I, Brouard J, Audrézet MP, Couderc Kohen L, Weiss L, Wizla N, Vrielynck S, LLerena K, Le Bourgeois M, Deneuville E, Remus N, Nguyen-Khoa T, Raynal C, Roussey M, Girodon E. Sermet-Gaudelus I, et al. Among authors: weiss l. Arch Pediatr. 2017 Apr;24(4):401-414. doi: 10.1016/j.arcped.2017.01.019. Epub 2017 Mar 1. Arch Pediatr. 2017. PMID: 28258861 French.
Guidelines for the clinical management and follow-up of infants with inconclusive cystic fibrosis diagnosis through newborn screening.
Sermet-Gaudelus I, Brouard J, Audrézet MP, Couderc Kohen L, Weiss L, Wizla N, Vrielynck S, LLerena K, Le Bourgeois M, Deneuville E, Remus N, Nguyen-Khoa T, Raynal C, Roussey M, Girodon E. Sermet-Gaudelus I, et al. Among authors: weiss l. Arch Pediatr. 2017 Dec;24(12):e1-e14. doi: 10.1016/j.arcped.2017.07.015. Epub 2017 Nov 22. Arch Pediatr. 2017. PMID: 29174009
Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis.
Burgel PR, Munck A, Durieu I, Chiron R, Mely L, Prevotat A, Murris-Espin M, Porzio M, Abely M, Reix P, Marguet C, Macey J, Sermet-Gaudelus I, Corvol H, Bui S, Lemonnier L, Dehillotte C, Da Silva J, Paillasseur JL, Hubert D; French Cystic Fibrosis Reference Network Study Group. Burgel PR, et al. Am J Respir Crit Care Med. 2020 Jan 15;201(2):188-197. doi: 10.1164/rccm.201906-1227OC. Am J Respir Crit Care Med. 2020. PMID: 31601120
Effect of one-year lumacaftor-ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients.
Misgault B, Chatron E, Reynaud Q, Touzet S, Abely M, Melly L, Dominique S, Troussier F, Ronsin-Pradel O, Gerardin M, Mankikian J, Cosson L, Chiron R, Bounyar L, Porzio M, Durieu I, Weiss L, Kessler R, Kessler L. Misgault B, et al. Among authors: weiss l. J Cyst Fibros. 2020 Sep;19(5):712-716. doi: 10.1016/j.jcf.2020.03.002. Epub 2020 Mar 19. J Cyst Fibros. 2020. PMID: 32201160 Free article.
2,985 results