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 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 ...
If you haven't heard of Rett syndrome, it's probably because it's very rare. Affecting an estimated one in 12,000 baby girls ...
The root causes of most forms of Alzheimer's disease largely remain a mystery. Now, researchers have revealed a new piece of ...
Biomedical engineers at Duke University have demonstrated a promising new approach that could be used to treat a rare and ...
Accumulation of clots causes some of the most severe symptoms of sickle cell disease, including strokes, kidney failure, ...
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 ...
Worryingly low levels of genetic diversity make Scotland’s red squirrels especially vulnerable to disease, a study has shown.
Jared Corbett was born with down syndrome. He has worked at a grocery store for the last 23-years. But he has another talent, ...