Sarepta Therapeutics, Inc.
Antisense-induced exon2 inclusion in acid alpha-glucosidase

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Abstract:

The present disclosure relates to antisense oligomers and related compositions and methods for inducing exon inclusion as a treatment for glycogen storage disease type II (GSD-II) (also known as Pompe disease, glycogenosis II, acid maltase deficiency (AMD), acid alpha-glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to inducing inclusion of exon 2 and thereby restoring levels of enzymatically active acid alpha-glucosidase (GAA) protein encoded by the GAA gene.

Status:
Grant
Type:

Utility

Filling date:

5 Sep 2014

Issue date:

1 Feb 2022