At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

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

Sarepta Therapeutics Inc. said another patient has died from acute liver failure after receiving one of its gene therapies, putting additional pressure on the biotech company after the recent deaths ...

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 ...

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

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

Sarepta Therapeutics is facing a challenging period after the deaths of two patients who received its Columbus-invented gene ...

This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

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 ...

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 ...

On a conference call, CEO Doug Ingram said the decision to cut 36% of the company's staff and halt several drug programs was ...