为期 5 年的 Evrysdi 研究证实了其对 1 型 SMA 儿童的有效性和安全性，其中 91% 存活，81% 脱离通气，59% 无需支撑即可坐着。 5-year Evrysdi study confirms efficacy and safety for Type 1 SMA children, with 91% alive, 81% off ventilation, and 59% sitting without support.
罗氏 Evrysdi 研究的 5 年数据证实了其对患有 1 型脊髓性肌萎缩症 (SMA) 的儿童的疗效和安全性。 5-year data from Roche's Evrysdi study confirms its efficacy and safety for children with Type 1 spinal muscular atrophy (SMA). 第五年结束时，91% 的儿童还活着，81% 的儿童无需永久性通气，59% 的儿童无需支撑即可坐着。 At the end of Year 5, 91% of children were alive, 81% without permanent ventilation, and 59% could sit without support. 目前，Evrysdi 已在 100 多个国家获得批准，全球已有超过 15,000 名患者接受治疗。 Over 15,000 patients have been treated globally, with Evrysdi now approved in 100+ countries.