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Ionis Pharmaceuticals公司公布Zilganersen在亚历山大病试验中取得的显著成果

-- Ionis Pharmaceuticals (IONS) 表示,一项研究表明,其 50 毫克剂量的 zilganersen 对患有亚历山大病(一种罕见且通常致命的神经退行性疾病)的 5 岁及以上患者取得了具有统计学意义的显著疗效。 该公司周二在一份声明中表示,这项关键性研究达到了其主要终点,该药物在 10 米步行测试中显示出具有临床意义的步速稳定作用。 该公司表示,来自 Gross Motor Function Measure-88 的新数据显示,接受 zilganersen 治疗的 2 至 4 岁儿童的粗大运动功能改善程度高于研究的对照组。 Ionis 表示,zilganersen 展现出良好的安全性和耐受性,大多数不良事件的严重程度为轻度或中度。 Ionis 表示,该药物正在接受美国食品药品监督管理局 (FDA) 的优先审查,预计 FDA 将于 9 月 22 日前做出批准决定。

Price: $75.13, Change: $+0.25, Percent Change: +0.34%

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