Stoke Therapeutics, Inc.
ANTISENSE OLIGOMERS FOR TREATMENT OF AUTOSOMAL DOMINANT MENTAL RETARDATION-5 AND DRAVET SYNDROME

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.