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• W4323306376 abstract "Treatment options for inherited metabolic epilepsies are rapidly expanding with advances in molecular biology and the genomic revolution. Traditional dietary and nutrient modification and inhibitors or enhancers of protein and enzyme function, the mainstays of therapy, are undergoing continuous revisions to increase biological activity and reduce toxicity. Enzyme replacement and gene replacement and editing hold promise for genetically targeted treatment and cures. Molecular, imaging, and neurophysiologic biomarkers are emerging as key indicators of disease pathophysiology, severity, and response to therapy." @default.
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• W4323306376 date "2023-03-06" @default.
• W4323306376 modified "2023-09-24" @default.
• W4323306376 title "New Therapeutic Approaches to Inherited Metabolic Pediatric Epilepsies" @default.
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• W4323306376 doi "https://doi.org/10.1212/wnl.0000000000207133" @default.
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