Over the past decade, fragile X syndrome (FXS) has been at the forefront of translational efforts in neurodevelopmental disorders to bring targeted treatments from basic laboratory studies and animal models to patients and their families, with the goal of normalizing the neurobiology, behavioral disturbances and cognitive deficits associated with the Fragile X Mental Retardation 1 (FMR1) gene mutation. Here, FMR1 is linked to fragile X syndrome.