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Ionis Pharmaceuticals公司公佈Zilganersen在亞歷山大病試驗中取得的顯著成果

-- Ionis Pharmaceuticals (IONS) 表示,一項研究表明,其 50 毫克劑量的 zilganersen 對患有亞歷山大病(一種罕見且通常致命的神經退化性疾病)的 5 歲及以上患者取得了具有統計學意義的顯著療效。 該公司週二在一份聲明中表示,這項關鍵性研究達到了其主要終點,該藥物在 10 公尺步行測試中顯示出具有臨床意義的步速穩定作用。 該公司表示,來自 Gross Motor Function Measure-88 的新數據顯示,接受 zilganersen 治療的 2 至 4 歲兒童的粗大運動功能改善程度高於研究的對照組。 Ionis 表示,zilganersen 展現出良好的安全性和耐受性,大多數不良事件的嚴重程度為輕度或中度。 Ionis 表示,該藥物正在接受美國食品藥物管理局 (FDA) 的優先審查,預計 FDA 將於 9 月 22 日前做出批准決定。

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