Vinay Prasad is returning to his role at the U.S. Food and Drug Administration to resume overseeing vaccine, gene therapy and ...

Vinay Prasad is returning to his role at the U.S. Food and Drug Administration, a little more than a week after he had left ...

The FDA approved labeling changes for elivaldogene autotemcel (eli-cel; Skysona), a one-time gene therapy for patients with ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, ...

Shares of drugmaker Sarepta Therapeutics surged in afterhours trading Monday after the company said it would resume shipping ...

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

If successful, the access model for sickle cell disease gene therapies could serve as a blueprint for other such treatments ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development ...

U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a ...