A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

Fakeeh University Hospital is emerging as a global destination for advanced gene therapy, delivering highly specialized ...

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 ...

A baby born with a rare and life-threatening genetic disorder has taken his first steps after receiving a groundbreaking, ...

JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...

Spark Therapeutics’ experimental gene therapy, Luxturna, improves vision in patients with a rare, inherited form of blindness, according to an initial review by Food and Drug Administration. Here are ...

Sarepta Therapeutics is still assessing how best to prevent liver injury from its commercialized Duchenne muscular dystrophy gene therapy. But Sarepta partner Hansa Biopharma has encouraging ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a ...

The US FDA has approved the first ever cell-based gene therapy to treat Wiskott-Aldrich syndrome (WAS).