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., ...

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

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

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

Sarepta Therapeutics shares surged 34% in after-hours trading on Wednesday after the company said it would slash 500 jobs, or ...

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 ...

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.

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

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

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

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