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110 results
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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: peltz sw. J Clin Pharmacol. 2007 Apr;47(4):430-44. doi: 10.1177/0091270006297140. J Clin Pharmacol. 2007. PMID: 17389552 Clinical Trial.
Ataluren as an agent for therapeutic nonsense suppression.
Peltz SW, Morsy M, Welch EM, Jacobson A. Peltz SW, et al. 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.
Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.
Shieh PB, Mcintosh J, Jin F, Souza M, Elfring G, Narayanan S, Trifillis P, Peltz SW, Mcdonald CM, Darras BT; THE ACT DMD STUDY GROUP. Shieh PB, et al. Among authors: peltz sw. Muscle Nerve. 2018 Nov;58(5):639-645. doi: 10.1002/mus.26191. Epub 2018 Sep 27. Muscle Nerve. 2018. PMID: 30028519 Free PMC article. Clinical Trial.
Ataluren treatment of patients with nonsense mutation dystrophinopathy.
Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, Connolly AM, Day JW, Flanigan KM, Goemans N, Jones KJ, Mercuri E, Quinlivan R, Renfroe JB, Russman B, Ryan MM, Tulinius M, Voit T, Moore SA, Lee Sweeney H, Abresch RT, Coleman KL, Eagle M, Florence J, Gappmaier E, Glanzman AM, Henricson E, Barth J, Elfring GL, Reha A, Spiegel RJ, O'donnell MW, Peltz SW, Mcdonald CM; PTC124-GD-007-DMD STUDY GROUP. Bushby K, et al. Among authors: peltz sw. Muscle Nerve. 2014 Oct;50(4):477-87. doi: 10.1002/mus.24332. Muscle Nerve. 2014. PMID: 25042182 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: peltz sw. 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.
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. Among authors: peltz sw. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
The nucleoside analog clitocine is a potent and efficacious readthrough agent.
Friesen WJ, Trotta CR, Tomizawa Y, Zhuo J, Johnson B, Sierra J, Roy B, Weetall M, Hedrick J, Sheedy J, Takasugi J, Moon YC, Babu S, Baiazitov R, Leszyk JD, Davis TW, Colacino JM, Peltz SW, Welch EM. Friesen WJ, et al. Among authors: peltz sw. RNA. 2017 Apr;23(4):567-577. doi: 10.1261/rna.060236.116. Epub 2017 Jan 17. RNA. 2017. PMID: 28096517 Free PMC article.
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