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We herein report the case of a 15-year-old patient with MTHFR deficiency presenting with a slowly progressive mental decline and spastic paraplegia, followed by rapidly progressive psychosis and repetitive seizures triggered by infection that successfully responded to betaine treatment

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Preclinical and clinical safety data includes [5] : No evidence of mutagenic activity, chromosomal aberrations, or micronucleus formation in genotoxicity studies 6-month chronic oral administration studies in rats showed no toxicological concerns 9-month studies in cynomolgus monkeys revealed general safety Six human clinical trials spanning 0.25 mg to 30 mg doses for up to 6 months No serious adverse events or withdrawals related to AOD9604 intake No anti-AOD9604 antibodies detected, indicating low immunogenic potential No changes in vital signs, physical examinations, electrocardiograms, or safety-related laboratory parameters Research Applications A Quick Review AOD9604 is a peptide compound that has shown potential in research related to lipid metabolism, energy balance, and tissue regeneration