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2-Arylbenzo[d]oxazole Phosphinate Esters as Second-Generation Modulators of Utrophin for the Treatment of Duchenne Muscular Dystrophy.
Babbs A, Berg A, Chatzopoulou M, Davies KE, Davies SG, Edwards B, Elsey DJ, Emer E, Guiraud S, Harriman S, Lecci C, Moir L, Peters D, Robinson N, Rowley JA, Russell AJ, Squire SE, Tinsley JM, Wilson FX, Wynne GM. Babbs A, et al. J Med Chem. 2020 Jul 23;63(14):7880-7891. doi: 10.1021/acs.jmedchem.0c00807. Epub 2020 Jul 10. J Med Chem. 2020. PMID: 32551645
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.
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.
Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Tremblay JP, et al. Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. Mol Ther. 2013. PMID: 23369965 Free PMC article. No abstract available.
17 results