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NaBPC157 exhibited an anti-convulsant effect interacting with the GABAergic system and improved the diazepam efficacy when coadministered (10 g/kg, 10 ng/kg i.p.) with diazepam (5.0 mg/kg i.p., twice daily for 10 days)

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Given the role of LoF mutations in ASD etiology, many of the therapeutics in development are designed to rescue the haploinsufficient effect of genes at the transcriptional, translational, and protein levels