{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["8(12)"],"submitter":["Erickson CA"],"pubmed_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."],"journal":["Brain sciences"],"pagination":["E224"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC6315698"],"repository":["biostudies-literature"],"pubmed_title":["Best Practices in Fragile X Syndrome Treatment Development."],"pmcid":["PMC6315698"],"pubmed_authors":["Lachiewicz A","Abbeduto L","Miller RM","Kaufmann WE","Weber JD","Erickson CA","Hessl D","Berry-Kravis E","Budimirovic DB","Haas-Givler B","Hagerman RJ"],"additional_accession":[]},"is_claimable":false,"name":"Best Practices in Fragile X Syndrome Treatment Development.","description":"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.","dates":{"release":"2018-01-01T00:00:00Z","publication":"2018 Dec","modification":"2024-10-18T20:46:12.798Z","creation":"2019-03-26T22:36:49Z"},"accession":"S-EPMC6315698","cross_references":{"pubmed":["30558274"],"doi":["10.3390/brainsci8120224"]}}