The FDA called for Sarepta Therapeutics to stop shipping its muscular dystrophy gene therapy, but the company said no.

Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 ...

The death of a third patient tied to a Sarepta Therapeutics gene therapy this year sent the company’s shares nosediving 36% Friday, halting the momentum the stock generated just a day earlier after ...

Scientists have made major progress in treating congenital deafness using gene therapy. This approach repairs hearing loss by fixing the root cause—a faulty gene known as OTOF. The OTOF gene creates ...

Drugmaker Sarepta Therapeutics said late Friday it will not comply with a request from the Food and Drug Administration to ...

Sarepta Therapeutics lays off 493 workers amid FDA probe, stock drop, and concerns over its gene therapy treatment, Elevidys.

Fermented stevia with banana leaf bacteria kills pancreatic cancer cells in lab tests. A compound called CAME triggers cell death.