17.5 rocket phase 2a trial: safety, tolerability, and efficacy of ov101 (gaboxadol) in adolescents and young adult males with fragile x syndrome
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.
Source: Journal of the American Academy of Child and Adolescent Psychiatry - Category: Psychiatry Authors: Dejan B. Budimirovic, Kelli C. Dominick, Lidia V. Gabis, Maxwell Adams, Mathews Adera, Linda Huang, Pamela Ventola, Nicole Tartaglia, Elizabeth Berry-Kravis Source Type: research
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