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 ...
Managed Healthcare Executive9h
Gene Therapies for Rare Diseases Twice as Likely to Win FDA Nod, Study Finds
None of the cell and gene therapies that reached the final regulatory submission stage were rejected by the FDA.
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, ...
This internationally recognized NJ doctor is on the frontlines battling rare diseases
A rare disease in the US is one in 1,500, or fewer than 200,000 people, and an ultra-rare disease is one in 50,000 or fewer ...
Medical Dialogues14h
USFDA grants orphan drug designation to Sanofi Rilzabrutinib for two rare diseases
Paris: Sanofi has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to ...
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 ...
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.
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 ...
ScienceAlert on MSN8d
Drug For Rare Disease Turns Human Blood Into Mosquito Poison
Scientists have a radical new plan for controlling mosquito numbers and fighting malaria: lacing human blood with a drug that ...
Mother with rare ALS touts 'miracle drug' that has stopped her disease
Raziel Green, 52, an active runner and mother of two, was diagnosed with a rare form of ALS in 2017. Since starting a ...