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

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

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

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

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 ...

Ocugen says it is on track to reshape the market for gene therapies against eye disorders over the next three years, by ...

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

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

From innovation in manufacturing to more-flexible regulation and better communication with payers, much needs to happen to ...

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

Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune ...