Best practices in fragile X syndrome treatment development
© 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.
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
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