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TGN-073 is an orally active, blood-brain barrier-permeable small molecule facilitator (positive allosteric modulator) of aquaporin-4 (AQP4), originally developed at Niigata University. By enhancing AQP4-mediated water transport, TGN-073 increases interstitial fluid circulation and glymphatic system clearance in the brain. In preclinical studies, TGN-073 has demonstrated therapeutic potential by reducing amyloid-beta accumulation in models of Alzheimer's disease, improving social behavior and restoring excitation/inhibition balance in models of Duchenne muscular dystrophy (Dp140 deficiency), and exhibiting analgesic effects in pain models. It is also widely utilized as a pharmacological research tool to investigate glymphatic transport and water diffusivity.
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