Sarepta Therapeutics, Inc.
MODIFIED ANTISENSE OLIGOMERS FOR EXON INCLUSION IN SPINAL MUSCULAR ATROPHY
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Abstract:
The present disclosure relates to modified antisense oligomers and related compositions and methods for increasing the expression of functional SMN protein and methods for treating spinal muscular atrophy and relates to inducing inclusion of exon 7 in SMN2 mRNA.
Status:
Application
Type:
Utility
Filling date:
23 Nov 2020
Issue date:
10 Jun 2021