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206 results

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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.
Clancy JP, Rowe SM, Accurso FJ, Aitken ML, Amin RS, Ashlock MA, Ballmann M, Boyle MP, Bronsveld I, Campbell PW, De Boeck K, Donaldson SH, Dorkin HL, Dunitz JM, Durie PR, Jain M, Leonard A, McCoy KS, Moss RB, Pilewski JM, Rosenbluth DB, Rubenstein RC, Schechter MS, Botfield M, Ordoñez CL, Spencer-Green GT, Vernillet L, Wisseh S, Yen K, Konstan MW. Clancy JP, et al. Among authors: accurso fj. Thorax. 2012 Jan;67(1):12-8. doi: 10.1136/thoraxjnl-2011-200393. Epub 2011 Aug 8. Thorax. 2012. PMID: 21825083 Free PMC article. Clinical Trial.
Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation.
Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW, Donaldson SH, Moss RB, Pilewski JM, Rubenstein RC, Uluer AZ, Aitken ML, Freedman SD, Rose LM, Mayer-Hamblett N, Dong Q, Zha J, Stone AJ, Olson ER, Ordoñez CL, Campbell PW, Ashlock MA, Ramsey BW. Accurso FJ, et al. N Engl J Med. 2010 Nov 18;363(21):1991-2003. doi: 10.1056/NEJMoa0909825. N Engl J Med. 2010. PMID: 21083385 Free PMC article. Clinical Trial.
Optimizing nasal potential difference analysis for CFTR modulator development: assessment of ivacaftor in CF subjects with the G551D-CFTR mutation.
Rowe SM, Liu B, Hill A, Hathorne H, Cohen M, Beamer JR, Accurso FJ, Dong Q, Ordoñez CL, Stone AJ, Olson ER, Clancy JP; VX06-770-101 Study Group. Rowe SM, et al. Among authors: accurso fj. PLoS One. 2013 Jul 26;8(7):e66955. doi: 10.1371/journal.pone.0066955. Print 2013. PLoS One. 2013. PMID: 23922647 Free PMC article. Clinical Trial.
Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.
Kerem E, Konstan MW, De Boeck K, Accurso FJ, Sermet-Gaudelus I, Wilschanski M, Elborn JS, Melotti P, Bronsveld I, Fajac I, Malfroot A, Rosenbluth DB, Walker PA, McColley SA, Knoop C, Quattrucci S, Rietschel E, Zeitlin PL, Barth J, Elfring GL, Welch EM, Branstrom A, Spiegel RJ, Peltz SW, Ajayi T, Rowe SM; Cystic Fibrosis Ataluren Study Group. Kerem E, et al. Among authors: accurso fj. Lancet Respir Med. 2014 Jul;2(7):539-47. doi: 10.1016/S2213-2600(14)70100-6. Epub 2014 May 15. Lancet Respir Med. 2014. PMID: 24836205 Free PMC article. Clinical Trial.
Biomarkers for cystic fibrosis drug development.
Muhlebach MS, Clancy JP, Heltshe SL, Ziady A, Kelley T, Accurso F, Pilewski J, Mayer-Hamblett N, Joseloff E, Sagel SD. Muhlebach MS, et al. J Cyst Fibros. 2016 Nov;15(6):714-723. doi: 10.1016/j.jcf.2016.10.009. Epub 2016 Oct 27. J Cyst Fibros. 2016. PMID: 28215711 Free PMC article. Review.
Long term effects of denufosol tetrasodium in patients with cystic fibrosis.
Ratjen F, Durham T, Navratil T, Schaberg A, Accurso FJ, Wainwright C, Barnes M, Moss RB; TIGER-2 Study Investigator Group. Ratjen F, et al. Among authors: accurso fj. J Cyst Fibros. 2012 Dec;11(6):539-49. doi: 10.1016/j.jcf.2012.05.003. Epub 2012 Jun 8. J Cyst Fibros. 2012. PMID: 22682898 Free article. Clinical Trial.
Standard care versus protocol based therapy for new onset Pseudomonas aeruginosa in cystic fibrosis.
Mayer-Hamblett N, Rosenfeld M, Treggiari MM, Konstan MW, Retsch-Bogart G, Morgan W, Wagener J, Gibson RL, Khan U, Emerson J, Thompson V, Elkin EP, Ramsey BW; EPIC; ESCF Investigators. Mayer-Hamblett N, et al. Pediatr Pulmonol. 2013 Oct;48(10):943-53. doi: 10.1002/ppul.22693. Epub 2013 Jul 2. Pediatr Pulmonol. 2013. PMID: 23818295 Free PMC article. Clinical Trial.
206 results