US FDA expands approval for Alnylam's drug to treat rare heart disease
U.S. Food and Drug Administration approved Alnylam's drug to treat a rare and deadly heart disease on Thursday, allowing the ...
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 ...
Country music icon retiring with rare disease. Here’s what to know
He took the stage and was great last week at the Grand Ole Opry’s 100th anniversary show, and now he is down to just two ...
BioSpace2d
Novartis’ Fabhalta Approved as First Therapy for Rare Kidney Disease
This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal ...
My son’s rare disease diagnosis spurred me to ditch Big Pharma for a biotech startup
Most individuals diagnosed with a rare disease won’t live to become teenagers or adults. I knew what I had to do.
Polar plunge raises thousands for John Hudson Dilgen, Staten Islander with rare skin disease
Dilgen, a 22-year-old Tottenville resident, has been battling epidermolysis bullosa (EB), a rare, life-threatening genetic ...
American Council on Science and Health9d
“Rare” but Not Uncommon: The Hidden Crisis of Rare Diseases
Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...
Prince Frederik of Luxembourg dies from rare disease
Prince Frederik of Luxembourg, the youngest son of Prince Robert of Luxembourg and Princess Julie of Nassau, has died after a ...
What is PolG? The rare genetic disease that ended a young prince's life
Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more awareness to the rare condition that impacted his life.
Meet Daniela, the 12-year-old girl battling a rare disease who’s become the UConn women’s secret weapon
When the UConn women’s basketball team takes the court, they know that no matter what happens, they’ve got the support of one ...
Cherokee Phoenix9d
Cherokee Nation Rare Disease Task Force outlines recommendations tribe can take to assist those with rare diseases
The Cherokee Nation Health Services Rare Disease Task Force has released a report outlining progress made over the past year ...