Compositions and methods for treating muscular dystrophies
AssigneeThe Regents of the University of California
InventorsRachelle H. Crosbie, Cynthia Shu, Varghese John, Jesus Campagna, Barbara Jagodzinska, Liubov Parfenova, Ekaterina Mokhonova
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Abstract
The present disclosure relates to compounds that increase sarcospan expression. The disclosure further relates to methods of treating a disease related to diminution or dysfunction of a dystrophin-related complex in a subject in need thereof.
Claims10
A compound represented by formula (IIb) or a pharmaceutically acceptable salt thereof:
- wherein
- X is O, N or NR9;
- R1, R2, R3, and R4 are each independently H, alkyl, alkoxy, halo, nitrile, amino or aminoalkyl;
- R5 is H, alkoxy, halo, nitrile, amino or aminoalkyl;
- R6 is H, ═O or alkyl;
- R9 is H or alkyl;
- and
- Y is O or NR10,
- R10 is H, alkyl or acyl;
- and
- m is 1 or 2.
A compound selected from:
- or a pharmaceutically acceptable salt thereof.
A method of treating a muscular dystrophy in a subject in need thereof, comprising administering to the subject a compound of formula (II) or a pharmaceutically acceptable salt thereof:
- wherein
- X is O, N or NR9;
- R1, R2, R3, R4 and R5 are each independently H, alkyl, alkoxy, halo, nitrile, amino or aminoalkyl;
- R6 is H, ═O or alkyl;
- Cy is aryl or heteroaryl;
- R7 and R8 are each independently H or alkyl, or taken together with the N atom to which they are attached form a heterocyclyl;
- and
- R9 is H or alkyl.
Dependencies are inferred from the claim text.