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Page 1
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: welch em. 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.
Nonsense-mediated mRNA decay in yeast.
Peltz SW, He F, Welch E, Jacobson A. Peltz SW, et al. Prog Nucleic Acid Res Mol Biol. 1994;47:271-98. doi: 10.1016/s0079-6603(08)60254-8. Prog Nucleic Acid Res Mol Biol. 1994. PMID: 8016322 Review. No abstract available.
Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL. Hirawat S, et al. Among authors: welch em. J Clin Pharmacol. 2007 Apr;47(4):430-44. doi: 10.1177/0091270006297140. J Clin Pharmacol. 2007. PMID: 17389552 Clinical Trial.
PTC124 targets genetic disorders caused by nonsense mutations.
Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL. Welch EM, et al. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
Nonsense suppression activity of PTC124 (ataluren).
Peltz SW, Welch EM, Jacobson A, Trotta CR, Naryshkin N, Sweeney HL, Bedwell DM. Peltz SW, et al. Among authors: welch em. Proc Natl Acad Sci U S A. 2009 Jun 23;106(25):E64; author reply E65. doi: 10.1073/pnas.0901936106. Epub 2009 Jun 8. Proc Natl Acad Sci U S A. 2009. PMID: 19506240 Free PMC article. No abstract available.
Targeting post-transcriptional control for drug discovery.
Peltz SW, Welch EM, Trotta CR, Davis T, Jacobson A. Peltz SW, et al. Among authors: welch em. RNA Biol. 2009 Jul-Aug;6(3):329-34. doi: 10.4161/rna.6.3.8953. Epub 2009 Jul 7. RNA Biol. 2009. PMID: 19574739 Free article. Review.
Ataluren as an agent for therapeutic nonsense suppression.
Peltz SW, Morsy M, Welch EM, Jacobson A. Peltz SW, et al. Among authors: welch em. Annu Rev Med. 2013;64:407-25. doi: 10.1146/annurev-med-120611-144851. Epub 2012 Nov 28. Annu Rev Med. 2013. PMID: 23215857 Free PMC article. Review.
46 results