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 ...
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 ...
Flow Space on MSN12h
What Is Stiff Person Syndrome, and What's It Like to Live With This Rare Condition?
Celine Dion is the best-known sufferer of this disorder, one of many autoimmune illnesses that disproportionately impact ...
NBC on MSN12h
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.
Australia and New Zealand are plagued by 'tall poppy syndrome'—but would a cure be worse than the disease?
The original tall poppies bloomed in the garden of Tarquin the Proud, last king of Rome. To communicate that his enemies ...
Pretzel Therapeutics Initiates Phase 1 Clinical Study Evaluating PX578, Lead Therapeutic in its Bioenergetics Restoration Franchise
Pretzel Therapeutics has initiated recruitment in a Phase 1 clinical study of PX578, the lead therapeutic in its bioenergetics restoration franchise.
Live Science19h
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, ...