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doi: 10.1172/JCI16521

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These inconsistent findings highlight the challenges in translating preliminary signals into robust clinical effects and indicate that, at present, there is insufficient evidence to support the routine use of arbaclofen in ASD (149–151).In another randomized, double-blind, placebo-controlled trial in children with FXS, ganaxolone—a GABAA receptor positive allosteric modulator with a favorable safety profile—failed to show significant improvement over placebo on the primary outcome (CGI-I) or the key secondary outcome (PARS-R) in the overall population