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

February is Rare Disease Month, an annual effort to highlight the challenges families can face while navigating rare ...

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

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

Accumulation of clots causes some of the most severe symptoms of sickle cell disease, including strokes, kidney failure, ...

More than 1,000 genes may serve as possible treatment targets for individuals with kidney disease, according to a new study, ...

Researchers at Washington University School of Medicine in St. Louis have conducted a longitudinal study on an individual ...

For the past month, local basketball programs have teamed up to raise awareness and spread hope for 9-year-old Autumn and ...

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 ...

Over 1,000 genes may serve as possible treatment targets for individuals with kidney disease, according to a new study. By creating the most complete and detailed genetic 'map' of kidney function to ...

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

In Europe alone, approximately 2 million people live with chronic inflammatory bowel diseases (IBD), and their incidence has ...