A loss of BAG3 leads to the accumulation of misfolded and damaged proteins, impairing the heart’s ability to contract. Rocket ...

Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) surged 15% after receiving a key regulatory boost from the U.S. Food and Drug ...

Sarepta Therapeutics will cut 500 jobs and add a serious warning on the label of its muscle-disorder gene therapy Elevidys, the drugmaker said on Wednesday, following the recent deaths of two patients ...

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...

IT之家 7 月 4 日消息，7 月 2 日，东南大学附属中大医院耳鼻咽喉头颈外科柴人杰教授，联合多家单位在国际顶级医学期刊 Nature Medicine 发表题为《AAV gene therapy for autosomal recessive ...

Sarepta Therapeutics faces a challenging period as it navigates recent setbacks with its groundbreaking gene therapy, leading ...

CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...

Sarepta Therapeutics said on Wednesday it would lay off 36% of its workforce, or about 500 employees, after the recent deaths of two patients who had received its gene therapy Elevidys.

An expert discusses how high-cost gene therapies require evidence standards including Phase 3 trial data showing complete response rates and durability, real-world evidence and health economic ...

The company is also pausing research for several treatments it has been developing for another form of muscular dystrophy.

Sarepta Therapeutics Inc. is cutting more than one-third of its workforce and will add a black box warning label to its gene therapy for a fatal muscle disorder after two patient deaths raised doubts ...

Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an excellent safety record. They provide evidence for the long-sought promise of gene therapy to deliver ...