This article provides compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SCN2A
RNA in cells or subjects, and in some cases, reducing the amount of SCN2A
protein in cells or subjects. Such compounds, methods, and pharmaceutical compositions can be used to improve at least one symptom or marker of a
disease or disorder associated with
voltage-gated
sodium channel proteins, such as, for example, developmental and epileptic
encephalopathy,
intellectual disability, or
autism spectrum disorder. Such symptoms and markers include, but are not limited to, seizures, hypotonia, sensory integration dysfunction, delayed and impaired motor development, intellectual and
cognitive impairment, motor and balance impairment,
visual impairment, language and speech
delay, gastrointestinal disorders, delayed neurodevelopment, sleep problems, and sudden unexpected death in
epilepsy.