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filingDate 2014-08-11-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2022-05-11-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber EP-3995580-A2
titleOfInvention Compounds and methods for modulation of dystrophia myotonica-protein kinase (dmpk) expression
abstract Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
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