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

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

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 ...

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

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

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

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 ...

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising ...

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

But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...