Sarepta Therapeutics, Inc.
EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
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Abstract:
Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 50 skipping are described. In various aspects, antisense oligomers are described according to Formula (I): ##STR00001## or a pharmaceutically acceptable salt thereof, wherein T, Nu, n, and R.sup.100 are defined herein.
Status:
Application
Type:
Utility
Filling date:
11 Jun 2021
Issue date:
20 Jan 2022