Sarepta Therapeutics, Inc.
COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY
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Abstract:
Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3'UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1.
Status:
Application
Type:
Utility
Filling date:
18 Oct 2018
Issue date:
29 Aug 2019