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EPX-100治疗Dravet综合征：II期研究已经启动

2020-09-18 Allan MedSci原创

Dravet综合征EPX-100
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Dravet综合征是一种罕见的儿童遗传性癫痫综合征，其特征是从婴儿期开始出现的难治性癫痫以及神经发育问题。70%-80%的DS患者检测出电压门控钠离子通道α-1亚基基因(SCN1A)突变。

Dravet综合征是一种罕见的儿童遗传性癫痫综合征，其特征是从婴儿期开始出现的难治性癫痫以及神经发育问题。70%-80%的DS患者检测出电压门控钠离子通道α-1亚基基因(SCN1A)突变。该综合征的典型特征是药物难治性癫痫发作，抗癫痫药物治疗的总体疗效有限。尽管如此，应尽可能避免发作诱因，并最大限度地控制发作和癫痫持续状态。药物治疗仍是主要治疗手段，部分患者也可选择生酮饮食和神经调控。

生物制药公司Epygenix Therapeutics近日宣布，该公司启动了EPX-100治疗Dravet综合征患儿的多中心、随机、双盲、安慰剂对照的概念验证试验。这项II期研究将证实EPX-100对Dravet综合征患儿的疗效和安全性。公司目前正在与国际CRO公司GreenLight Clinical合作招募患者。

Epygenix Therapeutics总裁兼首席执行官Hahn-Jun Lee博士表示：“我们对EPX-100 II期研究的启动感到非常兴奋，这是公司迈向下一阶段的里程碑”。

 

原始出处：

https://www.firstwordpharma.com/node/1758329?tsid=4

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    2020-10-04 yuanming7

    #EPX-100#

    0

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    2020-09-23 hulinmedical_52671245

    😯

    0

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    2020-09-20 zhang92560

    #Dravet综合征#

    0

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    2020-09-20 zhouqu_8

    #综合征#

    0

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