DMD是一种罕见且致命的遗传性疾病，由编码抗肌营养不良蛋白的DMD基因突变引起。这是一种不可逆的进行性疾病，会导致身体肌肉随着时间的推移而变得虚弱和受损，大多数患者在12岁之前就不得不依靠轮椅生活。在DMD患者中，约14%的患者体内已存在针对rh74 ...

Shares of Sarepta Therapeutics (NASDAQ:SRPT) rose on Wednesday after the market as it reported second quarter revenue and ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported ...

Sarepta Therapeutics (NASDAQ:SRPT) has hired a lobbying firm with close ties to the Trump administration as regulatory ...

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

2025年7月18日，FDA发布声明，要求Sarepta立即自愿停止Elevidys的全部商业供应。此前公司已主动停止对非行走患者的供药，但依然持续为尚能行走的患儿供药。面对FDA的请求，Sarepta选择拒绝，理由是“行走人群中并无新增或变化的安全 ...

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s request, which was issued Friday.

Sarepta Therapeutics’ stock was soaring Tuesday after the FDA recommended lifting the pause on the company’s Duchenne ...

Sarepta Therapeutics faces an "arduous path” to try to get its gene therapy for Duchenne muscular dystrophy back onto the ...

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...