Medics in the UK have become the first in the world to successfully administer a pioneering gene therapy that preserves sight in young children with an extremely rare genetic condition that usually ...

Researchers say amlodipine could serve as a safer option for patients without the risks that many other drugs carry In a ...

Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs ...

Researchers have debunked key beliefs about addiction, like that a person must hit "rock bottom" before having treatment or ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

Scientists have identified novel genetic interactions that may contribute to congenital heart disease (CHD), a common birth defect.

Scientists at the Icahn School of Medicine at Mount Sinai and collaborators have identified novel genetic interactions that may contribute to congenital heart disease (CHD), a common birth defect.

When a child might not be — when something is wrong, yet no one seems to know what it is — the anxiety is soul-crushing. And ...

MIT neuroscientists have made a breakthrough in treating fragile X syndrome by leveraging a novel neurotransmitter signaling ...

A study finds that lifestyle and environment influence health more than genetics. Smoking, income, and exercise shape disease risk.

Uterine cancer (also known as endometrial cancer) is the fourth most common cancer women in the U.S. are diagnosed with. It ...

Breast cancer is one of the most common types of cancer in women, according to the American Cancer Society. Although there ...