Best practices in fragile X syndrome treatment development


Journal Article

© 2018 by the authors. Licensee MDPI, Basel, Switzerland. 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.

Full Text

Duke Authors

Cited Authors

  • Erickson, CA; Kaufmann, WE; Budimirovic, DB; Lachiewicz, A; Haas-Givler, B; Miller, RM; Dixon Weber, J; Abbeduto, L; Hessl, D; Hagerman, RJ; Berry-Kravis, E

Published Date

  • December 1, 2018

Published In

Volume / Issue

  • 8 / 12

Electronic International Standard Serial Number (EISSN)

  • 2076-3425

Digital Object Identifier (DOI)

  • 10.3390/brainsci8120224

Citation Source

  • Scopus