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 ...
Your genes play a huge role in what makes and shapes your entire existence. Since you inherit your genes from your parents, ...
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 ...
Congenital heart disease (CHD) affects approximately 1% of live births. Thanks to surgical advancements, infants born with ...
Researchers at Washington University School of Medicine in St. Louis have conducted a longitudinal study on an individual ...
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...
More than 1,000 genes may serve as possible treatment targets for individuals with kidney disease, according to a new study, ...
Our study thus confirmed what was found in people of African descent in the US: the genetic origin underpinning the excess ...
A teenager was diagnosed with skin cancer and a brain tumour four times before she turned 18 after discovering she had a rare genetic disorder. Ruby-Jo Sneed discovered she had a rare genetic ...
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 ...