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

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 ...

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

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

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

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

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

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

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

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

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 ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...