US FDA approves expanded use of Amgen's rare disease drug
The U.S. Food and Drug Administration has approved expanded use of Amgen's drug, Uplizna, to help reduce the risk of flares ...
Live Science9h
This rare bacterial infection triggers pus-filled sores in the lungs and brain
Most cases of nocardiosis start out as lung infections, in which pus-filled cavities, or abscesses containing the bacteria, ...
EXCLUSIVE: How 1 dad used AI to advance finding a treatment for his son’s rare disease
AI helped Thomas Wagner understand Alexander disease, find connection with other conditions and discover researchers who ...
NBC on MSN2h
The Rare, Fatal GSS Prion Disease Was Just Featured on Chicago Med—What Is It?
After a devastating family loss, Chicago Med character Caitlin Lenox remains haunted by GSS, a rare, genetic prion disease.
US FDA approves Amgen's Uplizna as first treatment for rare immune disorder
The U.S. Food and Drug Administration has approved expanded use of Amgen's drug, Uplizna, to help reduce the risk of flares ...
Fairfax Co. teen turns rare disease diagnosis into youth-led support, advocacy
Khartik Uppalapati, 16, of Fairfax County, Virginia, has co-founded an advocacy group aimed at helping young people, like ...
Novel genes linked to rare childhood diarrhea disorder
At only two days old, Sophie was losing too much weight, and too quickly. Further genetic testing would show that Sophie has ...
Healio3h
Rilzabrutinib granted orphan drug designation for IgG4-related disease
The FDA has granted orphan drug designation to rilzabrutinib for IgG4-related disease and warm autoimmune hemolytic anemia, according to a press release from the manufacturer. There are currently no ...
My Son’s Disease Means I Know the Price of Trump’s Idiots’ ‘Mistakes’
My son is 8 and doctors are saving his life but worries we can go extinct from stupidity. He’s not wrong. News Editor Allison ...
'I thought I had a sinus infection - then doctors told me I have rare incurable disease'
After thinking he just had a stubborn sinus infection that wouldn't go away, Brian McNamara was told he had an ultra-rare ...
Live Science on MSN3d
In 'extremely rare' case, Michigan resident dies from rabies after receiving transplanted kidney carrying the virus
A Michigan resident has died from rabies following a kidney transplant at a hospital in Ohio. Investigations show that the ...
The National Institute for Health and Care Excellence2d
HST routing criteria: encouraging innovation in the treatment of ultra-rare diseases
Our chief medical officer, Professor Jonathan Benger, explains the crucial role the HST programme plays in encouraging ...