# US 10,500,223 B2 · Therapeutic agent for myotonic dystrophy

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- Number: US 10,500,223 B2
- Document ID: US10500223B2
- Status: Granted
- Kind: B2
- Application number: 15/743,622
- Filed: 2016-07-07
- Published: 2018-07-19
- Granted: 2019-12-10
- Earliest claimed: 2015-07-14
- Assignees: Osaka University
- Applicants: Osaka University
- Inventors: Masayuki Nakamori; Hideki Mochizuki
- Web page: https://patentgrep.com/patent/US10500223B2

## CPC

- A61K 31/7048: having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin {, digitoxin or digoxin}
- A61K 31/7052: having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
- 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 a therapeutic agent for myotonic dystrophy which inhibits aberrant splicing responsible for myotonic dystrophy, resulting in an increase in a normally spliced product and thus improvement in a symptom of myotonic dystrophy, and is highly safe for use in long-term administration. The therapeutic agent for myotonic dystrophy comprises, as an active ingredient, at least one compound selected from the group consisting of erythromycin, clarithromycin and azithromycin, a pharmaceutically acceptable salt or hydrate thereof, or a prodrug thereof.

## Claims

### Claim 1

A method for ameliorating at least one symptom of myotonic dystrophy in a mammal that has myotonic dystrophy, comprising:
selecting said mammal, which has myotonic dystrophy and presents at least one symptom of myotonic dystrophy, to receive a compound that ameliorates said at least one symptom of myotonic dystrophy; and
administering, to said mammal an effective amount of at least one compound selected from the group consisting of erythromycin, clarithromycin and azithromycin, a pharmaceutically acceptable salt or hydrate thereof, or a prodrug thereof, wherein
the at least one symptom of myotonic dystrophy is selected from the group consisting of muscular atrophy, muscle weakness, cardiac conduction defect, myocardiopathy, dementia, somnolence, cataract, retinal degeneration, impaired glucose tolerance and hyperlipidemia.

### Claim 2

A method for improving muscle contraction in a mammal that has myotonic dystrophy comprising:
selecting a mammal having myotonic dystrophy to receive an agent that improves muscle contraction; and
administering to said mammal a composition comprising at least one compound selected from erythromycin, clarithromycin or azithromycin, a pharmaceutically acceptable salt or hydrate thereof, or a prodrug thereof.