The death of a 16-year-old boy taking a gene therapy this month dealt a fresh blow to the Duchenne muscular dystrophy ...

这并非基因疗法首次遭遇致命危机，辉瑞公司宣布了该公司针对DMD的AAV9基因疗法PF-06939926的III期临床CIFFREO失败消息，该疗法曾造成两名患者死亡；另外，Elevidys在获批上市之前的Ⅲ期试验因未达主要终点引发疗效争议。而此次死亡 ...

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

“Elevidys会使肝脏某些酶水平升高，导致急性严重肝损伤，”处方说明书上指出。“急性肝病患者应暂停使用Elevidys，直到病情缓解或得到控制。对于既往存在肝功能损害或慢性肝炎病毒感染的患者，应谨慎考虑使用Elevidys。这些患者发生急性严重肝损伤的风险可能会增加。” ...

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.

Genetic medicine firm Sarepta Therapeutics announced the tragic death of a young man with Duchenne muscular dystrophy (DMD) ...

Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech ...

Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...