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