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Radiprodil is an investigational small molecule drug that acts as a selective and potent negative allosteric modulator of the N-methyl-D-aspartate (NMDA) receptor subunit 2B (NR2B or GluN2B). By binding to this subunit, radiprodil reduces NMDA receptor activity, which is implicated in excessive neuronal excitation and seizures. It has demonstrated antiseizure effects in preclinical models and early clinical studies, particularly for conditions characterized by enhanced GluN2B NMDA transmission such as GRIN-related neurodevelopmental disorders with gain-of-function mutations, tuberous sclerosis complex (TSC), focal cortical dysplasia (FCD) type II, and infantile spasms. Radiprodil has received FDA Orphan Drug Designation and Breakthrough Therapy designation for GRIN-related neurodevelopmental disorder with gain-of-function mutations[1][5][7].
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