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RST-401 is a discovery-stage small molecule being developed by ReST Therapeutics for the treatment of Rett Syndrome. It is part of a pipeline of precision neuropsychiatric candidates that target the GluN2D subunit of the NMDA receptor (NMDAR). The drug is designed to address NMDAR overactivation, which is implicated in the pathophysiology of Rett Syndrome. By selectively modulating or inhibiting GluN2D-containing receptors, particularly those on inhibitory interneurons, RST-401 aims to restore the balance of excitatory and inhibitory neurotransmission and provide neuroprotective effects.
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