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 ...

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

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

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

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.

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

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

Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.

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

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

The OUTREACH Study – Demonstrating that CAR-T therapies can be effectively administered in community cancer settings: In the ...

In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...