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.

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

Gene therapy has been headline news in recent years, in part due to the rapid development of biotechnology that enables doctors to administer such treatments. Broadly, gene therapies are ...

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

Since receiving gene therapy, Will Ungerer, shown in a recent picture, can do everyday things, including climb stairs easily and get dressed on his own. He’s also on a swim team. S. UNGERER ...

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.

Just getting selected to participate in the Bespoke Gene Therapy Consortium was a major hurdle. The eight diseases, including Talia's Charcot-Marie-Tooth disease type 4J or CMT4J, which causes ...