Year: 2018
Type: article
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.
Source: Brain Sciences
Institutions: Cincinnati Children's Hospital Medical Center, University of Cincinnati, Emory University, University of California, Davis, Kennedy Krieger Institute, Johns Hopkins University, Johns Hopkins Medicine, Duke University, Duke Medical Center, Autism & Developmental Medicine Institute, National Fragile X Foundation, Rush University Medical Center
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $2,654
OpenAlex ID: W2904556124