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9. 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 study. Hoppe JE; Chilvers M; Ratjen F; McNamara JJ; Owen CA; Tian S; Zahigian R; Cornell AG; McColley SA Lancet Respir Med; 2021 Sep; 9(9):977-988. PubMed ID: 33965000 [TBL] [Abstract][Full Text] [Related]
10. Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial. Sutharsan S; McKone EF; Downey DG; Duckers J; MacGregor G; Tullis E; Van Braeckel E; Wainwright CE; Watson D; Ahluwalia N; Bruinsma BG; Harris C; Lam AP; Lou Y; Moskowitz SM; Tian S; Yuan J; Waltz D; Mall MA; Lancet Respir Med; 2022 Mar; 10(3):267-277. PubMed ID: 34942085 [TBL] [Abstract][Full Text] [Related]
11. Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR. Rowe SM; McColley SA; Rietschel E; Li X; Bell SC; Konstan MW; Marigowda G; Waltz D; Boyle MP; Ann Am Thorac Soc; 2017 Feb; 14(2):213-219. PubMed ID: 27898234 [TBL] [Abstract][Full Text] [Related]
12. GLPG2737 in lumacaftor/ivacaftor-treated CF subjects homozygous for the F508del mutation: A randomized phase 2A trial (PELICAN). van Koningsbruggen-Rietschel S; Conrath K; Fischer R; Sutharsan S; Kempa A; Gleiber W; Schwarz C; Hector A; Van Osselaer N; Pano A; Corveleyn S; Bwirire D; Santermans E; Muller K; Bellaire S; Van de Steen O J Cyst Fibros; 2020 Mar; 19(2):292-298. PubMed ID: 31594690 [TBL] [Abstract][Full Text] [Related]
13. A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation. Davies JC; Sermet-Gaudelus I; Naehrlich L; Harris RS; Campbell D; Ahluwalia N; Short C; Haseltine E; Panorchan P; Saunders C; Owen CA; Wainwright CE; J Cyst Fibros; 2021 Jan; 20(1):68-77. PubMed ID: 32967799 [TBL] [Abstract][Full Text] [Related]
14. Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR. Donaldson SH; Pilewski JM; Griese M; Cooke J; Viswanathan L; Tullis E; Davies JC; Lekstrom-Himes JA; Wang LT; Am J Respir Crit Care Med; 2018 Jan; 197(2):214-224. PubMed ID: 28930490 [TBL] [Abstract][Full Text] [Related]
15. Clinical effect of lumacaftor/ivacaftor in F508del homozygous CF patients with FEV Aalbers BL; de Winter-de Groot KM; Arets HGM; Hofland RW; de Kiviet AC; van Oirschot-van de Ven MMM; Kruijswijk MA; Schotman S; Michel S; van der Ent CK; Heijerman HGM J Cyst Fibros; 2020 Jul; 19(4):654-658. PubMed ID: 31924546 [TBL] [Abstract][Full Text] [Related]
17. Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial. Davies J; Sheridan H; Bell N; Cunningham S; Davis SD; Elborn JS; Milla CE; Starner TD; Weiner DJ; Lee PS; Ratjen F Lancet Respir Med; 2013 Oct; 1(8):630-638. PubMed ID: 24461666 [TBL] [Abstract][Full Text] [Related]
18. Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. Middleton PG; Mall MA; Dřevínek P; Lands LC; McKone EF; Polineni D; Ramsey BW; Taylor-Cousar JL; Tullis E; Vermeulen F; Marigowda G; McKee CM; Moskowitz SM; Nair N; Savage J; Simard C; Tian S; Waltz D; Xuan F; Rowe SM; Jain R; N Engl J Med; 2019 Nov; 381(19):1809-1819. PubMed ID: 31697873 [TBL] [Abstract][Full Text] [Related]
19. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Southern KW; Murphy J; Sinha IP; Nevitt SJ Cochrane Database Syst Rev; 2020 Dec; 12(12):CD010966. PubMed ID: 33331662 [TBL] [Abstract][Full Text] [Related]
20. Tezacaftor/ivacaftor in people with cystic fibrosis who stopped lumacaftor/ivacaftor due to respiratory adverse events. Schwarz C; Sutharsan S; Epaud R; Klingsberg RC; Fischer R; Rowe SM; Audhya PK; Ahluwalia N; You X; Ferro TJ; Duncan ME; Bruinsma BG J Cyst Fibros; 2021 Mar; 20(2):228-233. PubMed ID: 32586736 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]