The U.S. Food and Drug Administration has approved expanded use of Amgen's drug, Uplizna, to help reduce the risk of flares ...

Most cases of nocardiosis start out as lung infections, in which pus-filled cavities, or abscesses containing the bacteria, ...

AI helped Thomas Wagner understand Alexander disease, find connection with other conditions and discover researchers who ...

A rare disease in the US is one in 1,500, or fewer than 200,000 people, and an ultra-rare disease is one in 50,000 or fewer ...

Khartik Uppalapati, 16, of Fairfax County, Virginia, has co-founded an advocacy group aimed at helping young people, like ...

The FDA has granted orphan drug designation to rilzabrutinib for IgG4-related disease and warm autoimmune hemolytic anemia, ...

After a devastating family loss, Chicago Med character Caitlin Lenox remains haunted by GSS, a rare, genetic prion disease.

After thinking he just had a stubborn sinus infection that wouldn't go away, Brian McNamara was told he had an ultra-rare ...

Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, ...

The FDA Wednesday approved the first drug to treat people with Prader-Willi syndrome, a rare genetic disease that causes an ...

A Michigan resident has died from rabies following a kidney transplant at a hospital in Ohio. Investigations show that the ...

Raziel Green, 52, an active runner and mother of two, was diagnosed with a rare form of ALS in 2017. Since starting a ...