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 ...

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

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.

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

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?

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 ...

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 ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College of Medicine and the UMass Chan Medical School have achieved ...

Editorial: Cutting-edge therapies exist, but the market cannot deliver them cheaply. Britain must build NHS capacity so that cures become collective goods, not expensive products ...