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Peer-Reviewed Publication
J Neurodev Disord2026;18(1)May 8, 2026Journal Article

Gaboxadol increases resting theta and alpha power without affecting evoked responses in fragile X syndrome in a home-based setting.

Lisa A De Stefano1, Hyeonseok Kim2, Craig A Erickson2,3, Lauren M Schmitt4, Kelli C Dominick2,3, Ernest V Pedapati2,3, Richard Huckle5, Robert Wilson5, Walker S McKinney6,7, Debra L Reisinger8,9, Meredith N Nelson8,9, Ashley E Dapore2, Paul S Horn9,10, Makoto Miyakoshi11,12
1Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. lisa.destefano@cchmc.org.
2Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
3Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
4Phelan-McDermid Syndrome Foundation, Osprey, FL, 34229, USA.
5HealX Ltd., Cambridge, UK.
6Division of Developmental and Behavioral Health, Children's Mercy Hospital, Kansas City, MO, USA.
7Department of Pediatrics, University of Missouri Kansas City School of Medicine, Kansas City, MO, USA.
8Division of Behavioral Medicine and Clinical Psychology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
9Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
10Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
11Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. makoto.miyakoshi@cchmc.org.
12Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine, Cincinnati, OH, USA. makoto.miyakoshi@cchmc.org.
PubMed PMC DOI PDF

Abstract

BACKGROUND: Fragile X syndrome (FXS) lacks FDA-approved treatments despite various small molecules contributing to phenotypic rescue in the FMR1 knockout (KO) mouse model. Translation from the mouse model has been hampered by phenotypic heterogeneity that contributes to participation barriers among participants who are most affected and may be unable to regularly visit the research laboratory. The…

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