Best practices in fragile X syndrome treatment development

Craig A. Erickson, Walter E. Kaufmann, Dejan B. Budimirovic, Ave Lachiewicz, Barbara Haas-Givler, Robert M. Miller, Jayne Dixon Weber, Leonard Abbeduto, David Hessl, Randi J. Hagerman, Elizabeth Berry-Kravis

Research output: Contribution to journalArticle

Abstract

Preclinical studies using animal models of fragile X syndrome have yielded several agents that rescue a wide variety of phenotypes. However, translation of these treatments to humans with the disorder has not yet been successful, shedding light on a variety of limitations with both animal models and human trial design. As members of the Clinical Trials Committee of the National Fragile X Foundation, we have discussed a variety of recommendations at the level of preclinical development, transition from preclinical to human projects, family involvement, and multi-site trial planning. Our recommendations are made with the vision that effective new treatment will lie at the intersection of innovation, rigorous and reproducible research, and stakeholder involvement.

Original languageEnglish (US)
Article number224
JournalBrain Sciences
Volume8
Issue number12
DOIs
Publication statusPublished - Dec 2018

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Keywords

  • Best practices
  • Clinical trials
  • Fragile X syndrome
  • Treatment development

ASJC Scopus subject areas

  • Neuroscience(all)

Cite this

Erickson, C. A., Kaufmann, W. E., Budimirovic, D. B., Lachiewicz, A., Haas-Givler, B., Miller, R. M., ... Berry-Kravis, E. (2018). Best practices in fragile X syndrome treatment development. Brain Sciences, 8(12), [224]. https://doi.org/10.3390/brainsci8120224