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.

主权项

1. A compound of Formula (I):wherein
m is 0, 1, 2, 3, or 4; each Rl is independently alkyl, cycloalkyl, aryl, heteroaryl, heterocycle, halo, hydroxyl, nitro, cyano, alkoxy, carboxy, trifluoromethyl, trifluoromethoxy, amino, or aminoalkyl; and each 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.