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

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

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 ...

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

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

AAVB-039 advances gene therapy for Stargardt disease, aiming for innovative treatments for inherited retinal disorders.

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

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

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

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

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 ...

- ZEVASKYN is the only FDA-approved therapy to treat RDEB wounds with a single application - ...