UCLA Health researchers identified EPAC2 as a potential drug target for Fragile X syndrome after work in genetically engineered Fmr1 knockout mice. The study, published in Neuron, reported that blocking EPAC2 restored abnormal brain activity patterns and improved multiple behavioral symptoms linked to Fragile X. Fragile X syndrome is caused by an FMR1 mutation leading to loss of FMRP, a key regulator of neuronal mRNA translation. Despite the monogenic basis that has made Fragile X a longstanding therapeutic candidate, no specific treatment has yet emerged as effective in clinical trials. The new work positions EPAC2 inhibition as a targeted strategy, supported by translatome profiling that highlighted opposing changes in inhibitory and excitatory neurons in the mouse model.