Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy.
EPILEPSIA(2023)
摘要
Ganaxolone was generally well tolerated and led to a greater reduction in the frequency of PCDH19-clustering seizures compared to placebo; however, the trend did not reach statistical significance. Novel trial designs are likely needed to evaluate the effectiveness of antiseizure treatments for PCDH19-clustering epilepsy.
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关键词
Encephalopathy,Ganaxolone,Pediatric,Safety,Seizures
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