US 12,109,204 B2
Methods for treating muscular dystrophies
Rachelle H. Crosbie, Sherman Oaks, CA (US); and Cynthia Shu, Oakland, CA (US)
Assigned to The Regents of the University of California, Oakland, CA (US)
Appl. No. 16/972,184
Filed by The Regents of the University of California, Oakland, CA (US)
PCT Filed Jun. 5, 2019, PCT No. PCT/US2019/035537
§ 371(c)(1), (2) Date Dec. 4, 2020,
PCT Pub. No. WO2019/236677, PCT Pub. Date Dec. 12, 2019.
Claims priority of provisional application 62/680,728, filed on Jun. 5, 2018.
Prior Publication US 2021/0361635 A1, Nov. 25, 2021
Int. Cl. A61K 31/4439 (2006.01); A61K 31/137 (2006.01); A61K 31/40 (2006.01); A61K 31/404 (2006.01); A61K 31/427 (2006.01); A61K 31/439 (2006.01); A61K 31/4418 (2006.01); A61K 31/4422 (2006.01); A61K 31/498 (2006.01); A61K 31/522 (2006.01); A61K 31/525 (2006.01); A61K 31/565 (2006.01); A61K 31/7076 (2006.01); A61P 21/06 (2006.01)
CPC A61K 31/4439 (2013.01) [A61K 31/137 (2013.01); A61K 31/40 (2013.01); A61K 31/404 (2013.01); A61K 31/427 (2013.01); A61K 31/439 (2013.01); A61K 31/4418 (2013.01); A61K 31/4422 (2013.01); A61K 31/498 (2013.01); A61K 31/522 (2013.01); A61K 31/525 (2013.01); A61K 31/565 (2013.01); A61K 31/7076 (2013.01); A61P 21/06 (2018.01)] 7 Claims
 
1. A method for treating or preventing a disease related to dysfunction of a dystrophin-related complex in a subject in need thereof, comprising administering to the subject a compound that increases the expression of sarcospan, wherein the compound is selected from: 2-[3-(1,3-Dihydro-1,3,3-trimethyl-2H-indol-2-ylidene)-1-propenyl]-3-ethyl-benzothiazolium iodide (AC-93253), 1-Azabicyclo[2.2.2]oct-3-yl acetate (aceclidine), 1H-benzo[g]pteridine-2,4-dione (alloxazine), methyl N-[(E)-(1-hydroxy-4-oxidoquinoxalin-4-ium-2-ylidene)methyl]iminocarbamate (carbadox), 4-[1-hydroxy-2-(propan-2-ylamino)ethyl]benzene-1,2-diol (isoproterenol), N-[(1-butylpyrrolidin-2-yl)methyl]-4-cyano-1-methoxynaphthalene-2-carboxamide (nafadotride), or a pharmaceutically acceptable salt or ester thereof.