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news.Marinus Pharmaceuticals has received $3m grant from the US Department of Defense for the study of Ganaxolone as a treatment of Fragile-X Syndrome (FXS).
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Scientific data suggests that Ganaxolone may be ideally suited to reverse FXS symptoms related to the down-regulation of the neurosteroid binding site on GABA-A ion channels.
The grant has been awarded to Randi Hagerman, Michael Rogawski and David Hessl of the University of California, Davis (UCD).
Further, the researchers expect to recruit 60 children between the ages of six and 17 years to study the safety and effectiveness of Ganaxolone for treating behaviors and anxiety common with FXS.
Marinus chief clinical and regulatory officer Gail Farfel said the fact that a study with Ganaxolone was awarded this grant is a testament to the potential for neurosteroid augmentation as a new treatment for FXS patients.