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 ...
Rare virus that killed Gene Hackman's wife linked to three deaths in California
A rare virus that killed actor Gene Hackman's wife in February has been linked to the deaths of three people in California.
Once-rare fungal diseases are killing millions in an unprepared world
Once considered obscure or opportunistic, invasive fungal infections are now surfacing with alarming frequency - and in ...
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 ...
Trump administration terminates advisory committee guiding states on newborn screening for rare genetic diseases
A federal advisory committee that provides guidance on which rare diseases newborns should be screened for has been dissolved ...
Fierce Pharma2d
Amgen wins key rare disease nod for Uplizna, checking off one goal in expansion push
With a label expansion for Uplizna pushing the drug into an untouched rare disease market, Amgen is getting its money’s worth ...
Rare disease paralyzes 3-year-old, parents urge vigilance
Three-year-old Logan Reese was diagnosed with transverse myelitis, a rare neurological condition caused by inflammation of ...
I felt lost after my dad was diagnosed with a rare neurological disease. Here's what I wish someone would have told me
"When you're a little girl, you think your daddy is going to be there until he's very old and grey — you don't imagine being ...
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 ...
Belfast Live on MSN13h
'I was diagnosed with rare disease at the age of 42 but I remain positive'
"Late onset FA is a strange mix of feeling sad about the diagnosis and worrying about the future, and feeling happy that I ...
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 ...
Star of hit HBO series says doctors once thought he might be dying from a rare disease
Comedian Adam Devine, who has starred in hit TV shows such as HBO’s “Righteous Gemstones” and ABC’s “Modern Family,” says he ...