The Urgent Need for Molecular Imaging to Confirm Target Engagement for Clinical Trials of Fragile X Syndrome and Other Subtypes of Autism Spectrum Disorder

Promising therapeutic agents for the symptoms in animal models of fragile X syndrome (FXS) have not resulted in similar advances in clinical trials of humans with FXS due to the dearth of tools to quantify their key cognitive and behavioral outcomemeasures with optimal validity and reliability. Therefore, experts strongly recommended an effort to develop and implement use of biomarkers in unfolding clinical trials in FXS. Molecular imaging provides a spectrum of agents to serve as biomarkers to confirm that humans with FXS exhibit the molecular abnormalities of animal models of FXS. Thus, molecular imaging provides the mechanism to establish target engagement in humans for clinical trials of novel agents for FXS.