Gene therapy is now being used for certain cancers and sickle cell anemia.A South Florida teen was among the first to get ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College ...

lt;pgt;Scientists have successfully reconstructed a virus thousands of years after it became extinct, a development they believe could herald a new step in treating genetic diseases such as cystic ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

WASHINGTON — For Wedam Minyila, hospital rooms have always meant blinding pain. “Like someone is jamming a knife in me,” he said. But for a brief moment on a recent December morning, Wedam, 19, who ...

A regimen of three immune-suppressing drugs may improve the effectiveness of gene therapy for people with DMD, research in ...