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 ...

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

After a second Elevidys patient death from acute liver failure, Sarepta in July launched a major restructuring that involves ...

Ophthalmology celebrates 50 years of groundbreaking innovations, highlighting anti-VEGF therapies, OCT imaging, and future ...

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 ...

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

In a groundbreaking development that could herald a new era in the treatment of inherited deafness, a recent clinical trial ...