发明名称 THERAPEUTIC METHODS AND AGENTS FOR TREATING MYOTONIC DYSTROPHY
摘要 The invention provides compounds, compositions and methods for treating myotonic dystrophy. The compounds can selectively bind to CUG repeats in RNA, or to CTG repeats in DNA, and inhibit replication of the nucleic acids.
申请公布号 US2014288080(A1) 申请公布日期 2014.09.25
申请号 US201414297037 申请日期 2014.06.05
申请人 Zimmerman Steven C.;Wong Chun-Ho;Hergenrother Paul J.;Peh Jessie 发明人 Zimmerman Steven C.;Wong Chun-Ho;Hergenrother Paul J.;Peh Jessie
分类号 C07D251/70 主分类号 C07D251/70
代理机构 代理人
主权项 1. A method for treating myotonic dystrophy comprising administering to a patient in need thereof an effective amount of a compound of Formula (I): wherein m is 0, 1, 2, 3, or 4;each R1 is independently alkyl, cycloalkyl, aryl, heteroaryl, heterocycle, halo, hydroxyl, nitro, cyano, alkoxy, carboxy, trifluoromethyl, trifluoromethoxy, amino, or aminoalkyl; andeach L is independently a divalent alkyl or a divalent radical of the formula —W-A-W— wherein each W is independently —N(R″)C(═O)—, —C(═O)N(R″)—, —OC(═O)—, —C(═O)O—, —S(O)—, —S(O)2—, —N(R″)—, —C(═O)—, —(CH2)n— where n is 1-10, or a direct bond; wherein each R″ is independently H, (C1-C6)alkyl, or a nitrogen protecting group; and A is (C1-C20)alkyl, (C2-C16)alkenyl, (C2-C16)alkynyl, (C3-C8)cycloalkyl, (C1-C20)heteroalkyl, (C6-C10)aryl, —(OCH2—CH2)n— where n is 1 to about 20, —C(O)NH(CH2)n wherein n is 1 to about 6, —N(Me)(CH2)n wherein n is 1 to about 6; or (C1-C20)alkyl, (C2-C16)alkenyl, (C2-C16)alkynyl, or —(OCH2—CH2)n— interrupted between two carbons, or between a carbon and an oxygen, with a heterocycle, or (C6-C10)aryl group;or a pharmaceutically acceptable salt thereof;wherein the administration thereby treats or reduces the symptoms of the myotonic dystrophy.
地址 Champaign IL US