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

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

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

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

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., ...

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

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

Sanofi has been granted a speedy review from the FDA for a gene therapy for geographic atrophy (GA), a complication of age-related macular degeneration (AMD), as it prepares to test it in a clinical ...

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 has named 33 states, as well as Washington, D.C., and Puerto Rico, as participants in its Cell and Gene Therapy Access Model, which is designed to treat ...

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