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34 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: almstead ng. J Clin Pharmacol. 2007 Apr;47(4):430-44. doi: 10.1177/0091270006297140. J Clin Pharmacol. 2007. PMID: 17389552 Clinical Trial.
Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy.
Woll MG, Qi H, Turpoff A, Zhang N, Zhang X, Chen G, Li C, Huang S, Yang T, Moon YC, Lee CS, Choi S, Almstead NG, Naryshkin NA, Dakka A, Narasimhan J, Gabbeta V, Welch E, Zhao X, Risher N, Sheedy J, Weetall M, Karp GM. Woll MG, et al. Among authors: almstead ng. J Med Chem. 2016 Jul 14;59(13):6070-85. doi: 10.1021/acs.jmedchem.6b00460. Epub 2016 Jul 6. J Med Chem. 2016. PMID: 27299569
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: almstead ng. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
Structure-activity relationship (SAR) optimization of 6-(indol-2-yl)pyridine-3-sulfonamides: identification of potent, selective, and orally bioavailable small molecules targeting hepatitis C (HCV) NS4B.
Zhang N, Zhang X, Zhu J, Turpoff A, Chen G, Morrill C, Huang S, Lennox W, Kakarla R, Liu R, Li C, Ren H, Almstead N, Venkatraman S, Njoroge FG, Gu Z, Clausen V, Graci J, Jung SP, Zheng Y, Colacino JM, Lahser F, Sheedy J, Mollin A, Weetall M, Nomeir A, Karp GM. Zhang N, et al. J Med Chem. 2014 Mar 13;57(5):2121-35. doi: 10.1021/jm401621g. Epub 2013 Dec 4. J Med Chem. 2014. PMID: 24266880
Discovery of novel HCV inhibitors: synthesis and biological activity of 6-(indol-2-yl)pyridine-3-sulfonamides targeting hepatitis C virus NS4B.
Zhang X, Zhang N, Chen G, Turpoff A, Ren H, Takasugi J, Morrill C, Zhu J, Li C, Lennox W, Paget S, Liu Y, Almstead N, Njoroge FG, Gu Z, Komatsu T, Clausen V, Espiritu C, Graci J, Colacino J, Lahser F, Risher N, Weetall M, Nomeir A, Karp GM. Zhang X, et al. Bioorg Med Chem Lett. 2013 Jul 1;23(13):3947-53. doi: 10.1016/j.bmcl.2013.04.049. Epub 2013 Apr 30. Bioorg Med Chem Lett. 2013. PMID: 23683597
Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects.
Kong R, Laskin OL, Kaushik D, Jin F, Ma J, McIntosh J, Souza M, Almstead N. Kong R, et al. Clin Pharmacol Drug Dev. 2019 Feb;8(2):172-178. doi: 10.1002/cpdd.645. Epub 2019 Jan 10. Clin Pharmacol Drug Dev. 2019. PMID: 30629861 Free PMC article. Clinical Trial.
Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F. Naryshkin NA, et al. Among authors: almstead ng. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390 Free article.
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