Table of Contents
Brain Sci., Volume 8, Issue 12 (December 2018)
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Cover Story (view full-size image) Targeted treatment development in fragile X syndrome (FXS) has experienced significant interest and [...] Read more. Targeted treatment development in fragile X syndrome (FXS) has experienced significant interest and associated resources allocation in the last decade. Despite this, no treatments are yet approved for FXS. This issue addresses many lessons learned from past efforts with an eye towards future approaches that will de-risk the FXS translational medicine pipeline. Such efforts will likely span new target identification, improved synchrony of preclinical to clinical evaluation of target engagement, improved treatment effect detection, and overall enhanced broad stakeholder input in the conduct of in particular large-scale trial efforts. The field remains hopeful that meaningful and significant treatment development opportunities remain and with improved development approaches overall success will be enhanced. View Paper here.