# US 12,109,204 B2 · Methods for treating muscular dystrophies

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- Number: US 12,109,204 B2
- Document ID: US12109204B2
- Status: Granted
- Kind: B2
- Application number: 16/972,184
- Filed: 2019-06-05
- Published: 2021-11-25
- Granted: 2024-10-08
- Earliest claimed: 2018-06-05
- Assignees: The Regents of the University of California
- Applicants: The Regents of the University of California
- Inventors: Rachelle H. Crosbie; Cynthia Shu
- Web page: https://patentgrep.com/patent/US12109204B2

## CPC

- A61K 31/4439: containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole (nicotine A61K31/465)
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61K 31/137: Arylalkylamines, e.g. amphetamine, epinephrine, salbutamol, ephedrine {or methadone}
- A61K 31/40: having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
- A61K 31/404: Indoles, e.g. pindolol
- A61K 31/427: not condensed and containing further heterocyclic rings
- A61K 31/428: condensed with carbocyclic rings
- A61K 31/439: the ring forming part of a bridged ring system, e.g. quinuclidine (8-azabicyclo [3.2.1] octanes A61K31/46)
- A61K 31/4418: having a carbocyclic group directly attached to the heterocyclic ring, e.g. cyproheptadine
- A61K 31/4422: 1,4-Dihydropyridines, e.g. nifedipine, nicardipine
- A61K 31/498: Pyrazines or piperazines ortho- and peri-condensed with carbocyclic ring systems, e.g. quinoxaline, phenazine
- A61K 31/519: ortho- or peri-condensed with heterocyclic rings
- A61K 31/522: having oxo groups directly attached to the heterocyclic ring, e.g. hypoxanthine, guanine, acyclovir
- A61K 31/525: Isoalloxazines, e.g. riboflavins, vitamin B2
- A61K 31/565: not substituted in position 17 beta by a carbon atom, e.g. estrane, estradiol
- A61K 31/7076: containing purines, e.g. adenosine, adenylic acid
- A61P 21/06: Anabolic agents (androgens A61P5/26)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

Provided herein are methods for treating and preventing a disease related to diminution or dysfunction of a dystrophin-related complex in a subject in need thereof, comprising administering to the subject a compound that increases sarcospan. Also provided herein are pharmaceutical compositions comprising a compound that increases sarcospan, or a pharmaceutically acceptable salt or ester thereof, useful for the treatments described herein.

## Claims

### Claim 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.

### Claim 2 (depends on 1)

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

### Claim 3 (depends on 2)

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.

### Claim 4 (depends on 2)

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

### Claim 5 (depends on 1)

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

### Claim 6 (depends on 1)

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

### Claim 7 (depends on 1)

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