EPX-100治疗Dravet综合征

2019-06-25 Allan MedSci原创

Epygenix是一家开发Dravet综合症精准药物的生物制药公司,近日宣布,已向FDA提交了EPX-100的研究性新药申请(IND)和快速通道申请,用于治疗Dravet综合征患者。

Epygenix是一家开发Dravet综合症精准药物的生物制药公司,近日宣布,已向FDA提交了EPX-100的研究性新药申请(IND)和快速通道申请,用于治疗Dravet综合征患者。在大多数情况下,该疾病是由SCN1A缺失引起的,SCN1A是一种编码脑电压门控钠通道的基因(Nav1.1)。对超过3500种化合物的筛选后,发现EPX-100能够在Dravet综合症的斑马鱼模型中消除自发性癫痫发作和惊厥行为。

Dravet综合症是一种罕见的、灾难性的、终生的癫痫形式,从出生后的第一年开始,伴有频繁或长期癫痫发作。随着疾病的进展,通常会观察到智力残疾、行为异常、步态和运动功能障碍以及死亡率增加。患者无法通过现有药物进行充分控制,并且伴随着SUDEP(不明原因的突然死亡)、癫痫相关事故(如溺水或感染)的风险增加。


原始出处:

http://www.firstwordpharma.com/node/1649135#axzz5rpYDX3hI

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