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

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 ...

This study marks the first time gene therapy has shown consistent success in both children and adults with OTOF-related ...

The FDA granted fast track designation to SAR446597, an intravitreal gene therapy for the treatment of geographic atrophy due to age-related macular degeneration, according to a press release from ...

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.

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 ...

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

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

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 ...

The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...