A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

The Food and Drug Administration on Thursday approved a treatment that uses gene therapy to treat severe hemophilia A, a rare and sometimes fatal blood disorder. The new drug, Roctavian, could save ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Experimental gene therapy studied in both China and the United States has allowed several children born with inherited deafness to hear. On Tuesday, the Children's Hospital of Philadelphia announced ...