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

Sarepta Therapeutics said on Monday it will resume shipping of its muscular disorder gene therapy to patients who can walk, ...

Sarepta Therapeutics shares snapped a three-day losing streak on Tuesday, as analysts said resumption of U.S. shipments for ...

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

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

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...

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

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

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

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

Ixo-vec reduced anti-VEGF injection frequency and was well tolerated in patients with wet AMD. A phase 3 pivotal trial ...

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