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, ...

US Food and Drug Administration Commissioner Marty Makary said he’s trying to persuade Vinay Prasad, the agency’s former head ...

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

A man born with Usher syndrome type 1b, a rare genetic disease that causes congenital deafness and progressive blindness, has ...

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

Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune ...

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

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

A groundbreaking study titled "An Engineered Intravitreal Injection Retinal-Pigment-Epithelium-Tropic Adeno-Associated Virus ...

For nearly three decades, Mayo Clinic researcher Christopher Evans, Ph.D., has pushed to expand gene therapy beyond its ...