Trials in adults with focal onset seizures and in children with infantile spasms have recently been completed. There are ongoing studies in patients with focal onset seizures, PCDH19 pediatric epilepsy, and behaviors in Fragile X syndrome.Ganaxolone has been shown to protect against seizures in animal models, and to act a positive allosteric modulator of the GABAA receptor. Clinical trials
The most common adverse events reported across clinical trials have been somnolence (sleepiness), dizziness, and fatigue. In 2015, the MIND Institute at the University of California, Davis, announced that it was conducting, in collaboration with Marinus Pharmaceuticals, a randomized, placebo-controlled, Phase 2 clinical trial evaluating the effect of ganaxolone on behaviors associated with Fragile X syndrome in children and adolescents. References
External links
"Ganaxolone". Drug Information Portal. U.S. National Library of Medicine.
History
AFM has only been formally tracked since 2014, since the incidence has spiked in recent years.A group in Texas reported having observed a pattern in 2013 of one to four cases per year with similar polio-like characteristics.In 2014, the CDC Morbidity and Mortality Weekly Report and a CDC Clinician Outreach and Communication Activity (COCA) conference call, noted that many cases had neck, back, or extremity pain, but otherwise those affected generally had normal sensation in their limbs. A few participants in the conference call discussed whether pain, later abating, might precede the onset of paralysis.An October 2014 report described outbreaks in California and Colorado, suggesting that the number of cases might be 100 or more nationwide. Diagnosis included a detailed medical history, MRI imaging, and the elimination of transverse myelitis or Guillain–Barré syndrome as potential causes. Physicians were using an online mailing list to communicate about similar cases in Alabama and Kansas. The largest known cluster of cases was in Colorado, with 29 total, 12 of whom had been reported from August and onwards of that year. Three of four cases treated in Alabama involved a complete inability to move one arm, reminiscent of peripheral nerve injury: The three cases since August really look like each other. They have severe arm flaccidity and no mental status changes. All of them have similar spine MRIs showing gray-matter involvement. You could lay all three MRIs on top of each other and they look almost the same. Its pretty striking. ...