Fragile X Syndrome (FX)

Patient Resources for

Fragile X Syndrome (FXS)

Fragile X Syndrome (FXS) is a genetic condition, caused by a mutation in the fmr1 gene, and is characterized by the development of a range of developmental problems, including cognitive impairment, learning disabilities, and behavioral challenges.

Ganaxolone and other agents that have been shown to improve GABA function in a mouse model and have also been shown to improve FXS symptoms in clinical trials. Ganaxolone may increase signaling at existing receptors to normalize GABA function, thereby reducing anxiety, hyperactivity, and other disabilities associated with this inherited disorder.

FXS is estimated to affect 100,000 children and adults in the U.S. Currently, there are no known cures or approved therapies for FXS.

How does FXS affect the body?

FXS can cause learning disabilities, developmental delays, and social or behavioral problems. Disabilities vary in severity, although symptoms are often milder in females than in males.

Where can I find more information on Fragile X Syndrome?

You can find additional educational and support resources on Fragile X Syndrome through the following foundations and patient advocacy groups:

Links to third party sites are provided for convenience purposes only. The information contained on these sites is not information provided, controlled or monitored by Marinus Pharmaceuticals in any way. Marinus Pharmaceuticals is not responsible in any way for the accuracy, completeness or fitness for any particular purpose of any content appearing on such sites.

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Clinical Development of Ganaxolone in Fragile X Syndrome

Ganaxolone was evaluated for the treatment of anxiety and attention in children with FXS in a Phase 2 exploratory clinical trial. Consistent with our expectations and ganaxolone’s mechanism of action, treatment with ganaxolone improved anxiety and hyperactivity across multiple measures in FXS patients with high baseline anxiety.

In addition, ganaxolone was generally safe and well tolerated.

Target Formulation

Liquid Suspension and Oral Dosing

The results from this study support the anxiolytic effect of ganaxolone and provide a strong rationale to advance the clinical development of ganaxolone in anxious FXS patients.

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Bibliography

Find publications referencing ganaxolone and its affect on seizures and neuropsychiatric disorders.

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