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 (C1-C20)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, benzyl, acetyl, or phenacyl; 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. |