Elevidis基因疗法显示,杜尚尼亚病人在3年以上的时间里取得了持久的运动功能增益,使疾病减慢了73%。 Elevidys gene therapy showed lasting motor function gains in Duchenne patients over 3 years, slowing disease by up to 73%.

萨雷普塔治疗公司报告说,其基因治疗Elevidys在杜申肌力发育不良患者的运动功能持续显著改善,治疗儿童的功能得分保持更高,与对照组相比,疾病进展速度减缓了高达73%. Sarepta Therapeutics reported that its gene therapy Elevidys showed sustained, significant improvements in motor function for Duchenne muscular dystrophy patients over three years, with treated children maintaining higher function scores and slowing disease progression by up to 73% compared to controls. 为4岁和4岁以上的流动病人核准的治疗已经用于全球1 200多个病人,没有新的安全顾虑。 The therapy, approved for ambulatory patients aged four and older, has been used in over 1,200 patients globally with no new safety concerns. 成果支持了持续发展,包括对非住院病人进行新的试验,并促进了强劲的财务业绩,在市场前贸易中的份额上升。 Results supported continued development, including a new trial for non-ambulant patients, and contributed to strong financial performance, with shares rising in premarket trading.