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Current preclinical research on therapeutic strategies and human clinical trials can be divided into drugs that target the primary cause, i.e., loss-of-function mutations in MECP2 , and drugs that target downstream consequences, including neurotransmitter receptor systems, neurotrophins, and their intracellular signaling pathways (Tables 3, 4)

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Research involving AOD-9604 centers on biochemical and structural analysis, including peptide sequence verification, molecular stability assessment, and investigational modeling of lipid-associated signaling pathways at the molecular level

Routes of Administration: Oral vs