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113 results

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A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.
Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Dřevínek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordoñez C, Elborn JS; VX08-770-102 Study Group. Ramsey BW, et al. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185. N Engl J Med. 2011. PMID: 22047557 Free PMC article. Clinical Trial.
Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis.
Rowe SM, Heltshe SL, Gonska T, Donaldson SH, Borowitz D, Gelfond D, Sagel SD, Khan U, Mayer-Hamblett N, Van Dalfsen JM, Joseloff E, Ramsey BW; GOAL Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network. Rowe SM, et al. Among authors: donaldson sh. Am J Respir Crit Care Med. 2014 Jul 15;190(2):175-84. doi: 10.1164/rccm.201404-0703OC. Am J Respir Crit Care Med. 2014. PMID: 24927234 Free PMC article.
Home Monitoring of Patients with Cystic Fibrosis to Identify and Treat Acute Pulmonary Exacerbations. eICE Study Results.
Lechtzin N, Mayer-Hamblett N, West NE, Allgood S, Wilhelm E, Khan U, Aitken ML, Ramsey BW, Boyle MP, Mogayzel PJ Jr, Gibson RL, Orenstein D, Milla C, Clancy JP, Antony V, Goss CH; eICE Study Team. Lechtzin N, et al. Am J Respir Crit Care Med. 2017 Nov 1;196(9):1144-1151. doi: 10.1164/rccm.201610-2172OC. Am J Respir Crit Care Med. 2017. PMID: 28608719 Free PMC article. Clinical Trial.
Building global development strategies for cf therapeutics during a transitional cftr modulator era.
Mayer-Hamblett N, van Koningsbruggen-Rietschel S, Nichols DP, VanDevanter DR, Davies JC, Lee T, Durmowicz AG, Ratjen F, Konstan MW, Pearson K, Bell SC, Clancy JP, Taylor-Cousar JL, De Boeck K, Donaldson SH, Downey DG, Flume PA, Drevinek P, Goss CH, Fajac I, Magaret AS, Quon BS, Singleton SM, VanDalfsen JM, Retsch-Bogart GZ. Mayer-Hamblett N, et al. Among authors: donaldson sh. J Cyst Fibros. 2020 Sep;19(5):677-687. doi: 10.1016/j.jcf.2020.05.011. Epub 2020 Jun 7. J Cyst Fibros. 2020. PMID: 32522463 Free PMC article. Review.
AZD5634, an inhaled ENaC inhibitor, in healthy subjects and patients with cystic fibrosis.
Kristensson C, Åstrand A, Donaldson S, Goldwater R, Abdulai R, Patel N, Gardiner P, Tehler U, Mercier AK, Olsson M, Ersdal E, Mäenpää J, Bramer T, Malmgren A, Bennett W, Keen C. Kristensson C, et al. J Cyst Fibros. 2022 Jul;21(4):684-690. doi: 10.1016/j.jcf.2022.02.010. Epub 2022 Feb 26. J Cyst Fibros. 2022. PMID: 35227647 Free article. Clinical Trial.
Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials.
Mayer-Hamblett N, Ratjen F, Russell R, Donaldson SH, Riekert KA, Sawicki GS, Odem-Davis K, Young JK, Rosenbluth D, Taylor-Cousar JL, Goss CH, Retsch-Bogart G, Clancy JP, Genatossio A, O'Sullivan BP, Berlinski A, Millard SL, Omlor G, Wyatt CA, Moffett K, Nichols DP, Gifford AH; SIMPLIFY Study Group. Mayer-Hamblett N, et al. Among authors: donaldson sh. Lancet Respir Med. 2023 Apr;11(4):329-340. doi: 10.1016/S2213-2600(22)00434-9. Epub 2022 Nov 4. Lancet Respir Med. 2023. PMID: 36343646 Free PMC article. Clinical Trial.
113 results