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104 results

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Page 1
Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective.
Mayer-Hamblett N, Clancy JP, Jain R, Donaldson SH, Fajac I, Goss CH, Polineni D, Ratjen F, Quon BS, Zemanick ET, Bell SC, Davies JC, Jain M, Konstan MW, Kerper NR, LaRosa T, Mall MA, McKone E, Pearson K, Pilewski JM, Quittell L, Rayment JH, Rowe SM, Taylor-Cousar JL, Retsch-Bogart G, Downey DG. Mayer-Hamblett N, et al. Among authors: donaldson sh. Lancet Respir Med. 2023 Oct;11(10):932-944. doi: 10.1016/S2213-2600(23)00297-7. Epub 2023 Sep 9. Lancet Respir Med. 2023. PMID: 37699421 Free PMC article. Review.
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: donaldson sh. 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: donaldson sh. 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.
Progress in cystic fibrosis and the CF Therapeutics Development Network.
Rowe SM, Borowitz DS, Burns JL, Clancy JP, Donaldson SH, Retsch-Bogart G, Sagel SD, Ramsey BW. Rowe SM, et al. Among authors: donaldson sh. Thorax. 2012 Oct;67(10):882-90. doi: 10.1136/thoraxjnl-2012-202550. Thorax. 2012. PMID: 22960984 Free PMC article.
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: donaldson sh. PLoS One. 2013 Sep 10;8(9):e73905. doi: 10.1371/journal.pone.0073905. eCollection 2013. PLoS One. 2013. PMID: 24040112 Free PMC article.
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.
A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial.
Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D; VX09-809-102 study group. Boyle MP, et al. Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24. Lancet Respir Med. 2014. PMID: 24973281 Clinical Trial.
Novel outcome measures for clinical trials in cystic fibrosis.
Tiddens HAWM, Puderbach M, Venegas JG, Ratjen F, Donaldson SH, Davis SD, Rowe SM, Sagel SD, Higgins M, Waltz DA. Tiddens HAWM, et al. Among authors: donaldson sh. Pediatr Pulmonol. 2015 Mar;50(3):302-315. doi: 10.1002/ppul.23146. Epub 2014 Dec 30. Pediatr Pulmonol. 2015. PMID: 25641878 Free PMC article. Review.
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.
104 results