Ganaxolone has been employed in clinical trials, with the enrollment of 605 participants in six clinical trials, with a focus on drug-resistant partial onset seizures, CDKL5 deficiency disorder, tuberous sclerosis, PCDH19-related epilepsy, status epilepticus, convulsive status epilepticus, non-convulsive status epilepticus, and epilepsy. The gene discussed is PCDH19; the disease is epilepsy.