Dijck, Anke Van

Ganaxolone Fragile X Clinical Trial Showed Disappointing Results

Hannah Maria Miles Dijck, Anke Van • Kooy, Frank • Research Updates • University of Antwerp, Belgium February 7, 2019February 12, 2019GABA, ganaxolone, mGluR, NIH

Ganaxolone, an experimental drug from Marinus Pharmaceuticals which targets GABA receptors, did not show promise for Fragile X syndrome in a clinical trial.

Clinical Trial of Ganaxolone in Patients with Fragile X Syndrome

FRAXA Research Foundation 2014 Grants • 2015 Grants • Clinical • Clinical Trials • Dijck, Anke Van • Has Results • Kooy, Frank • Research Grant Results • Treatment Targets • University of Antwerp, Belgium September 2, 2017March 9, 2020drug tolerance, GABA, GABA-A, ganaxolone, glutamate, seizure, sleep

With a $90,000 grant from FRAXA Research Foundation funded during 2014-2015, Dr. Frank Kooy and colleagues at the University of Antwerp are conducting a double blind crossover trial of ganaxolone in patients with Fragile X syndrome. Results of this study were mixed (see Marinus: Results from Phase 2 Exploratory Clinical Study Support Continued Development of Ganaxolone in Fragile X Syndrome.)