SCN2A regulatory compounds and methods

Abstract

To provide compounds and methods for modulating SCN2A. [Solution] The present invention provides compounds, methods, and pharmaceutical compositions that reduce the amount or activity of SCN2A RNA in cells or subjects, and in certain cases reduce the amount of SCN2A protein in cells or subjects. Such compounds, methods, and pharmaceutical compositions are useful for improving at least one symptom or feature of a disease or disorder related to voltage-gated sodium channel proteins, such as developmental and epileptic encephalopathy, intellectual disability, or autism spectrum disorder. Such symptoms and features include, but are not limited to, seizures, hypotonia, sensory integration disorders, delayed and impaired motor skills development, intellectual and cognitive impairment, motor and balance disorders, visual impairment, delayed language and speech, gastrointestinal disorders, delayed neurodevelopment, insomnia, and sudden, unexpected death in epilepsy.

Claims

[Claim 1] The invention described in the specification.

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