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Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy.
Chancellor DR, Davies KE, De Moor O, Dorgan CR, Johnson PD, Lambert AG, Lawrence D, Lecci C, Maillol C, Middleton PJ, Nugent G, Poignant SD, Potter AC, Price PD, Pye RJ, Storer R, Tinsley JM, van Well R, Vickers R, Vile J, Wilkes FJ, Wilson FX, Wren SP, Wynne GM. Chancellor DR, et al. Among authors: davies ke. J Med Chem. 2011 May 12;54(9):3241-50. doi: 10.1021/jm200135z. Epub 2011 Apr 15. J Med Chem. 2011. PMID: 21456623
Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse.
Tinsley JM, Fairclough RJ, Storer R, Wilkes FJ, Potter AC, Squire SE, Powell DS, Cozzoli A, Capogrosso RF, Lambert A, Wilson FX, Wren SP, De Luca A, Davies KE. Tinsley JM, et al. Among authors: davies ke. PLoS One. 2011 May 6;6(5):e19189. doi: 10.1371/journal.pone.0019189. PLoS One. 2011. PMID: 21573153 Free PMC article.
Second-generation compound for the modulation of utrophin in the therapy of DMD.
Guiraud S, Squire SE, Edwards B, Chen H, Burns DT, Shah N, Babbs A, Davies SG, Wynne GM, Russell AJ, Elsey D, Wilson FX, Tinsley JM, Davies KE. Guiraud S, et al. Among authors: davies ke, davies sg. Hum Mol Genet. 2015 Aug 1;24(15):4212-24. doi: 10.1093/hmg/ddv154. Epub 2015 May 1. Hum Mol Genet. 2015. PMID: 25935002 Free PMC article.
Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy.
Ricotti V, Spinty S, Roper H, Hughes I, Tejura B, Robinson N, Layton G, Davies K, Muntoni F, Tinsley J. Ricotti V, et al. PLoS One. 2016 Apr 7;11(4):e0152840. doi: 10.1371/journal.pone.0152840. eCollection 2016. PLoS One. 2016. PMID: 27055247 Free PMC article. Clinical Trial.
486 results