Best Practices in Fragile X Syndrome Treatment Development.
Ontology highlight
ABSTRACT:
SUBMITTER: Erickson CA
PROVIDER: S-EPMC6315698 | biostudies-literature | 2018 Dec
REPOSITORIES: biostudies-literature
Publications
Best Practices in Fragile X Syndrome Treatment Development.
Erickson Craig A CA Kaufmann Walter E WE Budimirovic Dejan B DB Lachiewicz Ave A Haas-Givler Barbara B Miller Robert M RM Weber Jayne Dixon JD Abbeduto Leonard L Hessl David D Hagerman Randi J RJ Berry-Kravis Elizabeth E
Brain sciences 20181215 12
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, transitio ...[more]
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