Abstract
This chapter is an up-to-date review of targeted treatment trials for patients with fragile X syndrome (FXS). Specifically, there is a focus on controlled trials directed toward cellular receptors and drugs, such as AMPA receptor activators, mGluR5 negative allosteric modulators, and GABA receptor agonists. While only some patients show improvement during these studies, advances in FXS animal models are helping to uncover specific biomarkers that may help to identify patients who will be the most responsive. Finally, this chapter discusses the need for future controlled trials, regarding quantitative outcome measures and biomarkers, targeted of treatment for FXS.
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