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[5] [6] Early-phase clinical trials including a Phase 2 trial published in the New England Journal of Medicine (2023) have reported significant reductions in body weight and improvements in metabolic markers, though this evidence remains preliminary and is not sufficient to establish a clinical dosing or reconstitution standard

A direct role for FMRP in activity-dependent dendritic mRNA transport links filopodial-spine morphogenesis to fragile X syndrome

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