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超70%患者获得缓解!别构小分子调节剂获FDA突破性疗法认定
这一突破性疗法认定的依据来自在携带GRIN基因功能获得性突变的小儿患者的1b期临床试验Honeycomb的积极数据。数据显示,radiprodil治疗的总体耐受性良好。符合条件的癫痫患者的中位可计数运动性癫痫发作(CMS)频率较基线减少了86%。在试验期间,71%的患者CMS减少超过50%,且7例患者中有6例在为期8周的维持期内至少80%的日子无癫痫发作。
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