ARYL DIAMIDINES AND PRODRUGS THEREOF FOR TREATING MYOTONIC DYSTROPHY
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United States of America Patent
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Issued Date -
Oct 24, 2013
app pub date -
Apr 19, 2013
filing date -
Apr 20, 2012
priority date (Note) -
Abandoned
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Abstract
Disclosed herein are compounds (for example, diamidine derivatives and prodrugs) and methods of use thereof, for example in treating muscular dystrophy (DM) or disease caused by a toxic RNA in a subject. In some embodiments, the methods include administering an effective amount of one of more of the disclosed compounds to a subject to treat or inhibit DM or a disease caused by or associated with toxic RNA, such as DM1, DM2, spinocerebellar ataxia type 8 (SCA8), fragile X tremor ataxia syndrome (FXTAS), or Huntington disease-like 2 (HLD2). In some examples, the methods include selecting a subject for treatment, for example selecting a subject with DM1, DM2, SCA8, FXTAS, or HLD2.
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