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Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809.
Van Goor F, Hadida S, Grootenhuis PD, Burton B, Stack JH, Straley KS, Decker CJ, Miller M, McCartney J, Olson ER, Wine JJ, Frizzell RA, Ashlock M, Negulescu PA. Van Goor F, et al. Among authors: ashlock m. Proc Natl Acad Sci U S A. 2011 Nov 15;108(46):18843-8. doi: 10.1073/pnas.1105787108. Epub 2011 Oct 5. Proc Natl Acad Sci U S A. 2011. PMID: 21976485 Free PMC article.
Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770.
Van Goor F, Hadida S, Grootenhuis PD, Burton B, Cao D, Neuberger T, Turnbull A, Singh A, Joubran J, Hazlewood A, Zhou J, McCartney J, Arumugam V, Decker C, Yang J, Young C, Olson ER, Wine JJ, Frizzell RA, Ashlock M, Negulescu P. Van Goor F, et al. Among authors: ashlock m. Proc Natl Acad Sci U S A. 2009 Nov 3;106(44):18825-30. doi: 10.1073/pnas.0904709106. Epub 2009 Oct 21. Proc Natl Acad Sci U S A. 2009. PMID: 19846789 Free PMC article.
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. Among authors: ashlock ma. 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.
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: ashlock ma. 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.
Multicenter intestinal current measurements in rectal biopsies from CF and non-CF subjects to monitor CFTR function.
Clancy JP, Szczesniak RD, Ashlock MA, Ernst SE, Fan L, Hornick DB, Karp PH, Khan U, Lymp J, Ostmann AJ, Rezayat A, Starner TD, Sugandha SP, Sun H, Quinney N, Donaldson SH, Rowe SM, Gabriel SE. Clancy JP, et al. Among authors: ashlock ma. PLoS One. 2013 Sep 10;8(9):e73905. doi: 10.1371/journal.pone.0073905. eCollection 2013. PLoS One. 2013. PMID: 24040112 Free PMC article.
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