The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

The National Academy of Inventors (NAI) today released the list of newly inducted fellows, and two Duke researchers have been ...

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...

A South Jersey college student with hemophilia B is now living a more normal life thanks to treatment developed from research ...

You’re reading an excerpt – sign up to get the whole newsletter in your inbox.Born in 1949, Chip Hailey is old enough to ...

Chicago's Lurie's Children's Hospital will expand care for patients living with rare genetic diseases and conditions thanks ...

Lurie Children's Hospital officially announced an expansion of research with the $11 million from Don and Anne Edwards, who ...