Methods for treating muscular dystrophies

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.

2. The method of claim 1 , wherein the disease related to dysfunction of a dystrophin-related complex is a muscular dystrophy.

3. The method of claim 2 , wherein the muscular dystrophy is Becker muscular dystrophy (BMD), congenital muscular dystrophy (CMD), Duchenne muscular dystrophy (DMD), distal muscular dystrophy, Emery-Dreifuss muscular dystrophy, facioscapulohumeral muscular dystrophy, limb-girdle muscular dystrophy, myotonic dystrophy, or oculopharyngeal muscular dystrophy.

4. The method of claim 2 , wherein the muscular dystrophy is Duchenne muscular dystrophy (DMD).

5. The method of claim 1 , whereby sarcospan mRNA transcript is increased.

6. The method of claim 1 , whereby sarcospan protein level is increased.

7. The method of claim 1 , wherein the subject is human.