Ganaxolone achieves primary endpoint in phase 3 trial for CDKL5 deficiency disorder, rare form of genetic epilepsy

RCT (n=101) showed a 32% reduction in 28-day major motor seizure frequency in patients receiving oral ganaxolone (a positive allosteric modulator of GABAA receptors) vs. 4% reduction for those receiving the placebo (p=0.002), at 17 weeks.

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Biospace Inc.