Sarepta Therapeutics (NASDAQ:SRPT) has hired a lobbying firm with close ties to the Trump administration as regulatory ...

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 ...

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

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

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

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

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

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

Choosing gene therapy Gray was in and out of hospitals seven times during each of the two years before her gene therapy. Then 32, she explained to her doctor that she was too tired to go on.

Gene therapy ‒ which has the potential to cure, not just treat disease ‒ has been approved so far only for very rare blood, neuromuscular and eye diseases, though more than 589 clinical trials ...

Another special investigational case, the first in humans, found that providing the mother with gene therapy while pregnant and continuing treatment after birth also prevented the devastating ...