Three 2026 Breakthrough Prize winners reflect on developing Luxturna, a gene therapy that treats blindness caused by rare ...

Saffie Sandford was born with Leber's Congenital Amaurosis and "missed out on a lot" due to difficulties seeing in any ...

Three Philadelphia scientists won a $3 million Breakthrough Prize for developing the first FDA-approved gene therapy for a genetic disease, prize sponsors announced Saturday. Their work created a ...

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 ...

The treatment, the first of its kind, was approved by the Food and Drug Administration on Thursday. “Our baby was born deaf, ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...