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 ...
Right now, approximately 20 billion red blood cells are busy traveling through your blood vessels. They are delivering oxygen ...
Researchers at Washington University School of Medicine in St. Louis have conducted a longitudinal study on an individual ...
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 ...
Our study thus confirmed what was found in people of African descent in the US: the genetic origin underpinning the excess ...
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 ...
Worryingly low levels of genetic diversity make Scotland’s red squirrels especially vulnerable to disease, a study has shown.
In the UK, approximately 17,500 people have sickle cell disease and 300 babies are born with the condition each year. It is a genetic disorder caused by inherited mutations in a person’s DNA ...