Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

The U.S. Food and Drug Administration said on Tuesday it has approved SpringWorks Therapeutics' drug to treat a type of rare ...

The Food and Drug Administration (FDA) approved a new drug to treat a disorder causing the growth of noncancerous tumors on ...

The root causes of most forms of Alzheimer's disease largely remain a mystery. Now, researchers have revealed a new piece of the puzzle.

Jared Corbett was born with down syndrome. He has worked at a grocery store for the last 23-years. But he has another talent, ...

A combination of human pedigree analysis and fruit fly genetics has identified a gene variant that appears to prevent PIGA-CDG, a rare but serious seizure disorder. The findings could ultimately lead ...

Health systems across the country are making it easier for primary care clinicians to order genetic tests for patients who ...