Drugmaker Sarepta Therapeutics said late Friday it won't comply with a request from the Food and Drug Administration to halt all shipments of its gene therapy following the death of a third ...

Gene therapy maker Sarepta tells FDA it won't halt shipments despite patient deaths Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments ...

Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk. Sarepta has reported three patient deaths related to its gene therapies.

Movies love a good distraction. While you’re watching the action, something subtle plays out in the corner. That quiet thing you barely noticed says more than the dialogue ever could.

Gene therapy can improve hearing in children and adults with congenital deafness or severe hearing impairment, a new study involving researchers at Karolinska Institutet reports.

A new gene therapy has shown promising results in restoring hearing to children, teens, and even a young adult born with a rare form of inherited deafness.

Gene therapy can improve hearing in children and adults with congenital deafness or severe hearing impairment, a new study involving researchers at Karolinska Institutet reports. Hearing improved ...

A retired pharmacist may have been cured from life threatening hemophilia B during "innovative" gene therapy treatment at Vanderbilt.

LAS VEGAS — Ashkan M. Abbey, MD, presented 1-year fellow eye substudy results for ABBV-RGX-314, a subretinal gene therapy from Regenxbio for wet age-related macular degeneration. At Clinical ...

The FDA has granted RMAT designation to detalimogene voraplasmid for the treatment of high-risk, BCG-unresponsive, NMIBC with CIS.

Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the death over the ...

Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for patients who can no longer walk, following the death of a second person who ...