Please note that Epilepsy Research UK does not endorse/promote individual epilepsy treatments or pharmaceutical companies.Marinus Pharmaceuticals has announced the results of a phase two clinical trial evaluating its drug candidate, ganaxolone, for the treatment of PCDH19 epilepsy. They show that ganaxolone was generally safe, well-tolerated and reduced seizure frequency in most participants.In a press release, Dr Michael G. Chez, Paediatric Neurologist at Sutter Medical Center in Sacramento, California, and an investigator in the trial said: “The benefit that ganaxolone provided in reducing seizures is clinically meaningful for these difficult-to-treat patients with a severe, rare epilepsy… In addition to seizure reduction, the patients that I treated with ganaxolone displayed improved behavior and cognitive skills during treatment.”“A drug that can lessen seizure burden and behavioral comorbidities caused by this disease would be welcomed by patients, their families and the medical community,” he added.The study enrolled 11 girls with PCDH19 epilepsy, aged 4 to 15 years. The children were first observed for a period of 12-weeks to assess their seizure frequency, and then given ganaxolone for 26 weeks.The results showed that seizure frequency was reduced in seven participants (64%) following treatment with ganaxolone. In addition, eight subjects (73%) had more seizure-free days during treatment with ganaxolone than during the 12-week period prior to the start of treatment.The company is now enrolling participants with other forms of paediatric genetic epilepsies, such as CDKL5 and Lennox-Gastaut Syndrome.PCDH19 epilepsy is a rare form of genetic epilepsy that mostly affects girls. It is characterised by early onset seizures and cognitive and behavioural difficulties.The condition is caused by a mutation in the PCDH19 gene, located on the X-chromosome. This causes the protein the gene encodes for, which supports communication between the cells of the nervous system, not to function properly or to be absent altogether.Although there are currently no approved drugs to treat this form of epilepsy, in 2015, the US Food and Drug Administration granted orphan drug designation to ganaxolone, which will help accelerate its development as a potential treatment.Author: Dr Özge ÖzkayaClick here for more articles about other treatments for epilepsy.