美国食品和药物管理局增加了Elevidys的严重肝脏风险警告,限制使用4岁以上的杜申肌肉缩症的男孩. FDA adds severe liver risk warning to Elevidys, restricts use to ambulatory boys age 4+ with Duchenne muscular dystrophy.

美国食品和药物管理局更新了Elevidys的标签, 附有急性肝衰竭和严重肝损伤的警告, 限制使用4岁及以上的杜申肌肉缩的门诊男孩. The FDA has updated Elevidys' label with a boxed warning for acute liver failure and serious liver injury, restricting its use to ambulatory boys aged 4 and older with Duchenne muscular dystrophy. 这一变化是在三人死亡之后发生的,包括两名非门诊病人死亡,促使这一群体从核准的指示中除名。 The change follows three deaths, including two in non-ambulatory patients, prompting removal of that group from the approved indication. 这种治疗现在针对某些DMD基因删除的病人,需要严格的监测和免疫抑制疗法。 The therapy is now contraindicated in patients with certain DMD gene deletions, and strict monitoring and immunosuppressive regimens are required. Sarepta已暂停运送给非门诊病人,并将研究一种新的免疫抑制方法,以便有可能恢复接触。 Sarepta has paused shipments to non-ambulatory patients and will study a new immunosuppression approach to potentially restore access.