The U.S. Food and Drug Administration approved Abeona Therapeutics' gene therapy for a rare skin disorder on Tuesday.

Millions of people around the world live with rare diseases, most of them caused by a misstep in their genes. Some conditions ...

Many diseases are caused by a missing or defective copy of a single gene. For decades, scientists have been working on gene ...

Martin Mwita, 20, is the first patient in St. Louis and among the first across the country to receive a new gene therapy for ...

These types of mistakes are often described as “never events,” a term first coined in 2002 to refer to preventable medical ...

Autism, also known as autism spectrum disorder, is a neurodevelopmental condition that affects how people communicate, behave ...

Brenan Cullimore had a rare genetic condition, Brugada syndrome, that led to cardiac arrest. His wife wants to raise ...

Valley parents are racing to find a treatment for their son's progressive genetic disorder that will eventually lead to him going blind. 'Big beautiful bill' could be 'most consequential piece of ...

WHS is a rare genetic disorder caused by the deletion of a segment of chromosome 4. This missing genetic material can result in various health and developmental issues. Newsweek spoke to Carlos ...

One Faribault, Minnesota, woman says every day is simply a "gift" as she's the only known person in the world with her unnamed genetic disorder. "I was told that she probably wouldn't make it past ...

Changes in brain connectivity before and after puberty may explain why some children with a rare genetic disorder have higher risk of developing autism or schizophrenia, according to a UCLA Health ...

A seminal study from researchers at the Icahn School of Medicine at Mount Sinai and their collaborators in the United Kingdom, Belgium, Spain, the Netherlands, and Iceland has uncovered a new genetic ...