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 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 ...
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, ...
Sanofi's Rilzabrutinib Gets FDA's Rare-Disease Drug Designation
Sanofi said that its Rilzabrutinib treatment was granted orphan drug designation, which is used in the U.S. by the Food and Drug Administration. The designation is a status assigned to a medicine ...
Healio8h
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 ...
What’s in that $9 billion the Trump administration is reviewing at Harvard?
The government has provided few specifics about how it arrived at the $9 billion sum, except to say that it is undertaking a ...
North Charleston child shares battle with rare Severe Aplastic Anemia diagnosis
A six-year-old diagnosed with a rare disease is sharing his experience in hopes that other kids like him will never have to ...
Trump Supports Proxy Voting for New Parents in Congress, a Blow to Johnson
The president’s comment was a rare instance in which he and House Speaker Mike Johnson were not on the same side of an issue.
News Medical on MSN18h
Genetic research offers hope for children with rare intestinal disorders
Further genetic testing would show that Sophie has one of a group of rare conditions called CODE (congenital diarrhea and enteropathies) that disrupts the function of cells in the intestine, causing ...