Drug for treating CKLD5 deficiency related epileptic seizures:ganaxolone
Ganaxolone is an oral GABAA receptor positive variant modulator developed by Marinas Pharmaceuticals in the United States,and it can specifically modulate GABAA receptors in the central nervous system to enhance temporal and tonic inhibition for seizure control.In March 2022,the U.S.Food and Drug Administration approved it as the first drug to treat cyclin-dependent kinase-like 5 deficiency based on the results of a major phase Ⅲ clinical trial.Clinical trials have shown that ganaxolone is well tolerated,and the treatment regimen based on this drug is effective in controlling seizure frequency of patients and improving their quality of life.
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