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Best Practices in Fragile X Syndrome Treatment Development

Craig A Erickson1,2, Walter E Kaufmann3,4, Dejan B Budimirovic5,6

  • 1Division of Child & Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA. craig.erickson@cchmc.org.

Brain Sciences
|December 19, 2018
PubMed

Insights

Fragile X syndrome treatments show promise in animal models but face translation challenges in humans. Recommendations focus on improving preclinical studies, human trials, and stakeholder engagement for effective therapies.

Area of Science:

  • Neuroscience
  • Genetics
  • Clinical Trials

Background:

  • Preclinical studies in animal models of fragile X syndrome (FXS) have identified potential therapeutic agents.
  • Translating these findings to human treatments for FXS has proven challenging due to limitations in current models and trial designs.

Purpose of the Study:

  • To discuss and provide recommendations for improving the development and translation of FXS treatments.
  • To address limitations in preclinical research, human trial design, and stakeholder involvement.

Main Methods:

  • Recommendations developed by the Clinical Trials Committee of the National Fragile X Foundation.
  • Discussion focused on preclinical development, transition to human studies, family engagement, and multi-site trial planning.

Main Results:

  • Identified limitations in the translation of preclinical findings to human therapies for FXS.
  • Proposed a framework for enhancing the rigor and reproducibility of research and clinical trials.

Conclusions:

  • Effective treatments for FXS require innovation, robust research, and active stakeholder involvement.
  • Recommendations aim to bridge the gap between preclinical discoveries and successful human therapeutic interventions for FXS.
Keywords:
best practicesclinical trialsfragile X syndrometreatment development

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