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Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein Translation.
Weetall M, Davis T, Elfring G, Northcutt V, Cao L, Moon YC, Riebling P, Dali M, Hirawat S, Babiak J, Colacino J, Almstead N, Spiegel R, Peltz SW. Weetall M, et al. Among authors: peltz sw. Clin Pharmacol Drug Dev. 2016 Jul;5(4):296-305. doi: 10.1002/cpdd.240. Epub 2016 Feb 2. Clin Pharmacol Drug Dev. 2016. PMID: 27310330 Free PMC article. Clinical Trial.
Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties.
Cao L, Weetall M, Trotta C, Cintron K, Ma J, Kim MJ, Furia B, Romfo C, Graci JD, Li W, Du J, Sheedy J, Hedrick J, Risher N, Yeh S, Qi H, Arasu T, Hwang S, Lennox W, Kong R, Petruska J, Moon YC, Babiak J, Davis TW, Jacobson A, Almstead NG, Branstrom A, Colacino JM, Peltz SW. Cao L, et al. Among authors: peltz sw. Mol Cancer Ther. 2019 Jan;18(1):3-16. doi: 10.1158/1535-7163.MCT-18-0863. Epub 2018 Oct 23. Mol Cancer Ther. 2019. PMID: 30352802 Free PMC article.
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.
Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform.
Cao L, Weetall M, Bombard J, Qi H, Arasu T, Lennox W, Hedrick J, Sheedy J, Risher N, Brooks PC, Trifillis P, Trotta C, Moon YC, Babiak J, Almstead NG, Colacino JM, Davis TW, Peltz SW. Cao L, et al. Among authors: peltz sw. PLoS One. 2016 Dec 16;11(12):e0168366. doi: 10.1371/journal.pone.0168366. eCollection 2016. PLoS One. 2016. PMID: 27992500 Free PMC article.
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
Identification of PTC725, an orally bioavailable small molecule that selectively targets the hepatitis C Virus NS4B protein.
Gu Z, Graci JD, Lahser FC, Breslin JJ, Jung SP, Crona JH, McMonagle P, Xia E, Liu S, Karp G, Zhu J, Huang S, Nomeir A, Weetall M, Almstead NG, Peltz SW, Tong X, Ralston R, Colacino JM. Gu Z, et al. Among authors: peltz sw. Antimicrob Agents Chemother. 2013 Jul;57(7):3250-61. doi: 10.1128/AAC.00527-13. Epub 2013 Apr 29. Antimicrob Agents Chemother. 2013. PMID: 23629699 Free PMC article.
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E; Clinical Evaluator Training Group; ACT DMD Study Group. McDonald CM, et al. Among authors: peltz sw. Lancet. 2017 Sep 23;390(10101):1489-1498. doi: 10.1016/S0140-6736(17)31611-2. Epub 2017 Jul 17. Lancet. 2017. PMID: 28728956 Clinical Trial.
The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines.
Luban J, Sattler RA, Mühlberger E, Graci JD, Cao L, Weetall M, Trotta C, Colacino JM, Bavari S, Strambio-De-Castillia C, Suder EL, Wang Y, Soloveva V, Cintron-Lue K, Naryshkin NA, Pykett M, Welch EM, O'Keefe K, Kong R, Goodwin E, Jacobson A, Paessler S, Peltz SW. Luban J, et al. Among authors: peltz sw. Virus Res. 2021 Jan 15;292:198246. doi: 10.1016/j.virusres.2020.198246. Epub 2020 Nov 26. Virus Res. 2021. PMID: 33249060 Free PMC article.
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: peltz sw. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390
112 results