Sarepta Therapeutics, Inc.
COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY
Last updated:
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:
5 Feb 2021
Issue date:
6 Jan 2022