Ionis Pharmaceuticals, Inc.
Compounds and Methods for Use in Dystrophin Transcript
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Abstract:
Provided herein are methods, compounds, and compositions for modulation of dystrophin pre-mRNA in an animal. Such methods, compounds, and compositions are useful, for example, to treat, prevent, or ameliorate one or more symptoms of Duchenne Muscular Dystrophy disease.
Status:
Application
Type:
Utility
Filling date:
18 Nov 2020
Issue date:
17 Mar 2022