# US 10,245,235 B2 · Compositions and methods for treating muscular dystrophy and other disorders

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- Number: US 10,245,235 B2
- Document ID: US10245235B2
- Status: Granted
- Kind: B2
- Application number: 15/842,580
- Filed: 2017-12-14
- Published: 2018-06-21
- Granted: 2019-04-02
- Earliest claimed: 2016-12-16
- Assignees: The Charlotte-Mecklenburg Hospital Authority
- Applicants: The Charlotte Mecklenburg Hospital Authority
- Inventors: Qi Long Lu
- Web page: https://patentgrep.com/patent/US10245235B2

## CPC

- A61K 31/047: having two or more hydroxy groups, e.g. sorbitol
- A61K 31/7004: Monosaccharides having only carbon, hydrogen and oxygen atoms
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- 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

The present invention provides compositions and methods of their use in treating muscular dystrophy and other disorders.

## Claims

### Claim 1

A method of treating a disorder associated with a mutation or loss of function in a fukutin related protein (FKRP) gene in a subject, comprising administering a therapeutically effective amount of a ribitol to a subject in need thereof daily for at least 30 days, thereby treating the disorder associated with a mutation or loss of function in a fukutin related protein (FKRP) gene in a subject, wherein the disorder associated with a mutation or loss of function in the FKRP gene is selected from the group consisting of limb-girdle muscular dystrophy (LGMD), Walker-Warburg syndrome (WWS), muscle-eye-brain disease (MEB), congenital muscular dystrophy (CMD), and any combination thereof.

### Claim 2 (depends on 1)

The method of claim 1 wherein administration of the therapeutically effective amount of the ribitol treats or reduces the development of muscle weakness.