Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

Children’s Hospital Los Angeles paused the use of Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy in the wake of rising safety concerns about liver toxicity, adding to ...

A standoff over Elevidys could have major consequences for Duchenne patients, gene therapy companies and the perception of ...

When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The ...

By Zachary Stieber Contributing Writer Sarepta Therapeutics will not agree to a regulatory request to pause shipments of a ...

ICER is listening to concerns like McSherry's, and Pearson says feedback from the Duchenne patient community is being incorporated into the organization's work.

The regulator had asked Sarepta Therapeutics to halt all shipments of its therapy, Ele­vidys, after three patients died from ...

Shares of Sarepta Therapeutics (NASDAQ: SRPT) plunged 26.85% in Friday pre-market after a clinical trial participant died ...

Finally, it seemed, Sarah Jenssen would have a chance to get a treatment that her family hoped could help her. A gene therapy for Sarah’s condition, Duchenne muscular dystrophy, won a wide ...

“With the benefit of newborn screening, the next generation of families will have even more time to evaluate, pursue and access FDA-approved Duchenne therapies while avoiding extensive surgeries ...