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 ...
3-yr-old dies waiting for rare disease funds from Centre
Kolkata: A three-year-old girl waiting for treatment for a rare disease died without getting any medical assistance under the ...
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 ...
Live Science14h
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, ...
Successful therapy confirmed for newborns with fatal metabolic disorder MoCD type A
The drug fosdenopterin/rcPMP considerably improves the chances of survival and developmental progress in infants with ...
Rare disease attacked Fairhaven woman's eyes, brain and kidneys. A neighbor saved her life.
Nellie Zygiel of Fairhaven has battled illness much of her life. Diagnosed with a rare disease, her life has been saved ...
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 ...
Managed Healthcare Executive5h
FDA Grants Accelerated Approval to Vanrafia for IgAN, a Rare Kidney Disease
Vanrafia reduces proteinuria in adults with primary immunoglobulin A nephropathy (IgAN). It has a wholesale acquisition cost ...
NBC on MSN7h
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.
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 ...
Zacks Investment Research on MSN8h
SNY's Rilzabrutinib Gets FDA's Orphan Drug Tag for Two Rare Diseases
Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, ...
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 ...