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Dalzanemdor is a novel, investigational small molecule drug that acts as a positive allosteric modulator of the NMDA (N-methyl-D-aspartate) receptor. It is an analogue of the neurosteroid 24(S)-hydroxycholesterol and was developed to enhance NMDA receptor activity, which is essential for learning, memory, and cognition. Dalzanemdor was primarily investigated for the treatment of cognitive impairment associated with neurological disorders such as Huntington's disease, Alzheimer's disease, Parkinson's disease, dementia, encephalitis, mild cognitive impairment, and Smith-Lemli-Opitz syndrome. The drug demonstrated good oral bioavailability and pharmacokinetic properties suitable for once-daily dosing in central nervous system disorders. Despite initial promise in preclinical models and early clinical trials showing safety and tolerability without inducing excitotoxicity or seizures, development has been discontinued after failing to meet efficacy endpoints in late-stage clinical trials[1][3][5][6][7].
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