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 ...
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 ...
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 ...
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 ...
NBC on MSN10h
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.
Biotech Alert: Searches spiking for these stocks today
These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include: ...
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Healio10h
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, ...
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 ...
How Milan's Whitten Dobbs survived rare, deadly triple infection to return to TSSAA baseball field
Milan sophomore Whitten Dobbs survived a health scare late last year. Now, he’s a big spark for the Bulldogs’ during the 2025 TSSAA baseball season.
Live Science16h
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, ...
Labiotech.eu19h
Beyond amyloid: The search for the next big thing in Alzheimer’s treatment
Delve into how the biotech industry is pivoting from the amyloid hypothesis to find new Alzheimer's disease treatments.
News-Medical.Net20h
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 ...