Fragile X syndrome (FXS), an inherited intellectual disability often associated with autism, is
caused by the loss of expression of the fragile X mental retardation protein. Tremendous
progress in basic, preclinical, and translational clinical research has elucidated a variety of
molecular-, cellular-, and system-level defects in FXS. This has led to the development of
several promising therapeutic strategies, some of which have been tested in larger-scale
controlled clinical trials. Here, we will summarize recent advances in understanding …