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

Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

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, ...

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

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

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 ...

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

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...

Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved ...

AAV gene therapy in early life provides lasting HIV protection in primates—offering hope for pediatric HIV intervention in ...