Abstract
Fragile X syndrome (FXS) is a rare genetic condition characterized by intellectual disability and behavioral dysfunction. There are currently no approved medications for the treatment of FXS. OV101 (gaboxadol) is a δ subunit-selective extrasynaptic GABAA receptor agonist that showed improvement in a mouse model of FXS and has been evaluated in a phase 2 study of Angelman syndrome. Here, we report on the safety, tolerability, and efficacy of OV101 from the ROCKET trial.
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Schedule a callMore From: Journal of the American Academy of Child & Adolescent Psychiatry
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