Stoke Therapeutics, Inc.
Antisense oligomers for treatment of autosomal dominant mental retardation-5 and Dravet Syndrome

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Abstract:

Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.

Status:
Grant
Type:

Utility

Filling date:

14 Dec 2016

Issue date:

10 Aug 2021