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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: miller ll. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
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: miller ll. J Clin Pharmacol. 2007 Apr;47(4):430-44. doi: 10.1177/0091270006297140. J Clin Pharmacol. 2007. PMID: 17389552 Clinical Trial.
Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.
Kerem E, Hirawat S, Armoni S, Yaakov Y, Shoseyov D, Cohen M, Nissim-Rafinia M, Blau H, Rivlin J, Aviram M, Elfring GL, Northcutt VJ, Miller LL, Kerem B, Wilschanski M. Kerem E, et al. Among authors: miller ll. Lancet. 2008 Aug 30;372(9640):719-27. doi: 10.1016/S0140-6736(08)61168-X. Epub 2008 Aug 20. Lancet. 2008. PMID: 18722008 Clinical Trial.
Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis.
Wilschanski M, Miller LL, Shoseyov D, Blau H, Rivlin J, Aviram M, Cohen M, Armoni S, Yaakov Y, Pugatsch T, Cohen-Cymberknoh M, Miller NL, Reha A, Northcutt VJ, Hirawat S, Donnelly K, Elfring GL, Ajayi T, Kerem E. Wilschanski M, et al. Among authors: miller ll, miller nl. Eur Respir J. 2011 Jul;38(1):59-69. doi: 10.1183/09031936.00120910. Epub 2011 Jan 13. Eur Respir J. 2011. PMID: 21233271 Free article. Clinical Trial.
Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
Sermet-Gaudelus I, Boeck KD, Casimir GJ, Vermeulen F, Leal T, Mogenet A, Roussel D, Fritsch J, Hanssens L, Hirawat S, Miller NL, Constantine S, Reha A, Ajayi T, Elfring GL, Miller LL. Sermet-Gaudelus I, et al. Among authors: miller ll, miller nl. Am J Respir Crit Care Med. 2010 Nov 15;182(10):1262-72. doi: 10.1164/rccm.201001-0137OC. Epub 2010 Jul 9. Am J Respir Crit Care Med. 2010. PMID: 20622033 Clinical Trial.
Ambulatory quantitative waking and sleeping cough assessment in patients with cystic fibrosis.
Kerem E, Wilschanski M, Miller NL, Pugatsch T, Cohen T, Blau H, Rivlin J, Shoseyov D, Reha A, Constantine S, Ajayi T, Hirawat S, Elfring GL, Peltz SW, Miller LL. Kerem E, et al. Among authors: miller ll, miller nl. J Cyst Fibros. 2011 May;10(3):193-200. doi: 10.1016/j.jcf.2011.02.003. Epub 2011 Apr 2. J Cyst Fibros. 2011. PMID: 21459051 Free article.
Patient-reported Outcomes and Muscle Strength after a Physiotherapy-led Exercise and Support Brace Intervention in Patients with Acute Injury of the Posterior Cruciate Ligament: A Two-year Follow-up Study.
Rasmussen RG, Jacobsen JS, Blaabjerg B, Nielsen TG, Miller LL, Lind M. Rasmussen RG, et al. Among authors: miller ll. Int J Sports Phys Ther. 2023 Aug 2;18(4):807-819. doi: 10.26603/001c.83214. eCollection 2023. Int J Sports Phys Ther. 2023. PMID: 37547839 Free PMC article.
414 results