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

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

Connecticut is now participating in a new federal program aimed at expanding treatment access for patients with sickle cell ...

In a groundbreaking development that could herald a new era in the treatment of inherited deafness, a recent clinical trial ...

Uppsala University Hospital-led investigators report that gene-edited donor islet cells survived 12 weeks inside a man with ...

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

Leveraging Northway Biotech’s 20+ years of CDMO expertise, Diorasis Therapeutics is advancing AAV gene therapy for glaucoma ...

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

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

Sarepta Therapeutics said on Monday it will resume shipping of its muscular disorder gene therapy to patients who can walk, ...

Twenty-three days after receiving the gene therapy, the boy first responded to someone calling out to him. "It was unbelievable — the best feeling," Yiyi told Fox News Digital.

The first gene therapy product approved by the Food and Drug Administration (a treatment for a form of leukemia, approved last summer) costs hundreds of thousands of dollars for each infusion.