生产企业：Stoke Therapeutics
Zorevunersen is a proprietary antisense oligonucleotide (ASO) that increases NaV1.1 protein expression by utilizing the healthy copy of the SCN1A gene, aiming to restore normal physiological NaV1.1 levels. This RNA-based approach modulates RNA activity without altering or inserting DNA, potentially reducing both seizure frequency and associated non-seizure comorbidities in Dravet syndrome.
共 1 项相关疾病
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