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Basmisanil is a highly selective small molecule negative allosteric modulator and inverse agonist of the α5 subunit-containing GABA A receptor. It was developed by Roche for the treatment of cognitive impairment associated with Down syndrome, as well as for other neurodevelopmental and neuropsychiatric disorders. Basmisanil acts by selectively inhibiting GABA-induced currents at α5-GABA A receptors, with minimal effect on other GABA A receptor subtypes. Preclinical studies demonstrated its potential to improve cognition without anxiogenic or proconvulsant effects, and clinical studies have shown good safety and tolerability in humans. The drug has been evaluated in Phase 2 trials for Down syndrome-related intellectual disability, schizophrenia-associated cognitive impairment, and is currently being investigated for Dup15q syndrome[1][6][7][8].
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