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 massive Danish study shows that most mental illnesses—like schizophrenia, bipolar disorder, and depression—occur in people with no close family history of the condition.

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

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

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

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

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

A third patient has died this year after getting a gene therapy from Sarepta Therapeutics Inc., raising questions about the ...

The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the ...

The drastic cost-cutting move follows the deaths of two teenagers that forced the company to restrict usage of its gene therapy for Duchenne muscular dystrophy.

On Sunday at 1 a.m. Eastern time, an announcement went out from the first-generation gene therapy company for Duchenne muscular dystrophy: A second patient who had been treated with Elevidys has ...

When you're diagnosing a child with Duchenne muscular dystrophy, says Alexandra Bonner, MD, of Cleveland Clinic, it's important to not only determine if she's a carrier of the gene mutation that ...