title: Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy.
abstract: A series of novel 2-arylbenzoxazoles that upregulate the production of utrophin in murine H2K cells, as assessed using a luciferase reporter linked assay, have been identified. This compound class appears to hold considerable promise as a potential treatment for Duchenne muscular dystrophy. Following the delineation of structure-activity relationships in the series, a number of potent upregulators were identified, and preliminary ADME evaluation is described. These studies have resulted in the identification of 1, a compound that has been progressed to clinical trials.
authors: Summit plc , 91 Milton Park, Abingdon, Oxfordshire, OX14 4RY, United Kingdom.
authors: Daniel R
authors: DR
authors: Chancellor
authors: Kay E
authors: KE
authors: Davies
authors: Olivier
authors: O
authors: De Moor
authors: Colin R
authors: CR
authors: Dorgan
authors: Peter D
authors: PD
authors: Johnson
authors: Adam G
authors: AG
authors: Lambert
authors: Daniel
authors: D
authors: Lawrence
authors: Cristina
authors: C
authors: Lecci
authors: Carole
authors: C
authors: Maillol
authors: Penny J
authors: PJ
authors: Middleton
authors: Gary
authors: G
authors: Nugent
authors: Séverine D
authors: SD
authors: Poignant
authors: Allyson C
authors: AC
authors: Potter
authors: Paul D
authors: PD
authors: Price
authors: Richard J
authors: RJ
authors: Pye
authors: Richard
authors: R
authors: Storer
authors: Jonathon M
authors: JM
authors: Tinsley
authors: Renate
authors: R
authors: van Well
authors: Richard
authors: R
authors: Vickers
authors: Julia
authors: J
authors: Vile
authors: Fraser J
authors: FJ
authors: Wilkes
authors: Francis X
authors: FX
authors: Wilson
authors: Stephen P
authors: SP
authors: Wren
authors: Graham M
authors: GM
authors: Wynne
mesh_terms: D000818:Animals; D001583:Benzoxazoles / Q000138:chemical synthesis* / Q000737:chemistry / Q000494:pharmacology; D002460:Cell Line; D051379:Mice; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D009281:Naphthalenes; D013329:Structure-Activity Relationship; D015854:Up-Regulation; D049411:Utrophin / Q000096:biosynthesis* / Q000235:genetics
chemical_list: D001583:Benzoxazoles; D009281:Naphthalenes; D049411:Utrophin; C559859:ezutromid