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However, combination protocols lack controlled clinical trials, and safety/efficacy data comes primarily from preclinical research and anecdotal reports

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In FXS, CGG repeat expansions in the FMR1 gene lead to the loss of FMRP, impairing the translation of GABAA receptor mRNAs, particularly the GABAA 5 subunit, reducing receptor density in the amygdala and nucleus accumbens, resulting in delayed language development and autistic-like behaviors (138)