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20250179503. Methods Us (Praxis Precision Medicines, .)

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METHODS OF USING OLIGOMERIC COMPOUNDS TO TREAT SCN2A-RELATED DISORDERS

Abstract: the human gene scn2a encodes human scn2a protein, the alpha-1 subunit of the voltage-gated sodium channel nav 1.2. mutations in scn2a are associated with a variety of neurodevelopmental and intellectual diseases and disorders, including developmental and epileptic encephalopathy (dee), such as early-onset dee. compositions and methods of using scn2a oligomeric compounds for treating scn2a related disorders, including early onset developmental and epileptic encephalopathy.

Inventor(s): Michael S. Oldham, Steven Petrou

CPC Classification: C12N15/1138 ({against receptors or cell surface proteins})

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