WEDNESDAY, March 19, 2025 -- A patient has died while receiving

2025年3月19日，星期三——一名患者在接收治疗时死亡

Elevidys

Elevidys

, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment.

，一种用于治疗杜氏肌营养不良症的基因疗法，标志着与该治疗相关的首例已知死亡病例。

The company behind the drug,

该药物背后的公司，

Sarepta Therapeutics

萨雷普塔治疗公司

, said that the patient suffered acute liver injury, a known side effect,

，称该患者患有急性肝损伤，这是已知的副作用，

The Associated Press

美联社

reported. However, the 'severity' of this case had not been seen before, the company noted. Elevidys has been used in more than 800 patients, the company stated.

据报道。然而，该公司指出，这种情况的“严重性”前所未见。该公司表示，Elevidys 已在 800 多名患者中使用。

Sarepta did not release details about the patient but said a recent infection may have played a role in the liver injury. The therapy has been used in more than 800 patients, the company said.

萨雷普塔公司没有透露该患者的详细信息，但表示最近的感染可能在肝损伤中起到了一定作用。该公司表示，这种疗法已经在800多名患者中使用过。

The company plans to update the prescribing information for Elevidys to reflect this case.

公司计划更新 Elevidys 的处方信息以反映此案例。

Shares of Sarepta, based in Cambridge, Massachusetts, dropped more than 27% on Tuesday, closing at about $73 per share.

总部位于马萨诸塞州剑桥的Sarepta公司股价周二下跌超过27%，收盘价约为每股73美元。

Elevidys, which costs $3.2 million for a one-time infusion, was the

Elevidys的一次性输液费用为320万美元，是

first gene therapy approved

首个基因疗法获批

in the U.S. for Duchenne muscular dystrophy, a rare condition that causes muscle weakness, mobility loss and early death in males.

在美国，杜氏肌营养不良症是一种罕见的疾病，会导致男性肌肉无力、行动能力丧失并早逝。

The FDA first granted the drug expedited approval in 2023, despite some scientists questioning its effectiveness.

尽管一些科学家质疑其有效性，FDA还是在2023年首次授予该药物快速批准。

In 2024, the agency expanded full approval to include patients 4 and older, regardless of mobility status.

2024年，该机构扩大了全面批准的范围，将4岁及以上的患者纳入其中，无论其行动能力如何。

The therapy works by using a disabled virus to insert a gene that helps the body produce dystrophin, a protein crucial for muscle function. Sarepta said more than 800 patients have received Elevidys so far,

该疗法通过使用一种无害的病毒插入一个帮助身体产生肌营养蛋白（一种对肌肉功能至关重要的蛋白质）的基因来发挥作用。萨雷普塔表示，迄今为止已有800多名患者接受了Elevidys治疗。

The Associated Press

美联社

reported.

已报告。

Sarepta has secured FDA accelerated approval for three other Duchenne drugs since 2016. But none have yet been confirmed to be effective and further studies are still ongoing.

自 2016 年以来，Sarepta 已获得 FDA 对其他三种 Duchenne 药物的加速批准。但目前尚未确认它们的有效性，进一步的研究仍在进行中。

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