Therapeutic agent for myotonic dystrophy
AssigneeOsaka University
InventorsMasayuki Nakamori, Hideki Mochizuki
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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.
Claims2
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.
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.
Dependencies are inferred from the claim text.