Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for ...

Sarepta Therapeutics on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy ...

Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death ...

Sarepta Therapeutics said it has temporarily suspended shipments of Elevidys® (delandistrogene moxeparvovec-rokl) for infusion in non-ambulatory patients following the second death in three months of ...

Gene therapy is revolutionizing how they're treated, and the Fred Hutch Cancer Center in Seattle is at the center of it all. ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

A single infusion of adeno-associated virus (AAV)-mediated gene therapy led to sustained clinical benefit and no late-onset ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

The world’s first patient to successfully receive a CRISPR gene-editing treatment was discharged from Children’s Hospital of Philadelphia on June 2, ABC News reported.  KJ Muldoon, a 10-month-old who ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...