New rare genetic disease affecting motor neuron and muscle control identified

An international research team, led by Shinghua Ding at the University of Missouri, has identified a previously unknown ...
NEWS10 ABC on MSN

Rare disease causing meat allergy sees ‘explosive rise’ across US: study

A once-rare disease that causes a person to develop a severe allergy to red meat is seeing an “explosive rise” across America ...
The Silicon Review

BridgeBio Reports Second Rare Disease Drug Success

BridgeBio reports second late-stage rare disease drug success, establishing new treatment potential for endocrine disorder ...

Experimental targeted therapy shows promise for histiocytosis, a rare blood cancer

The first time Joey Carlsen Martinez came to Memorial Sloan Kettering Cancer Center (MSK), he was in terrible shape. He had a ...

'We don’t have time': Parents of 4-year-old with rare disorder urge FDA to reconsider drug denial

The FDA denied approving DCA to treat a rare disorder called PDCD. The Higbees are urging the agency to reconsider.
USA Today

Her 5-year-old has a severe, rare disease. There's a cure, but she can't get it.

Catherine Illingworth, 39, knew something was wrong the moment her son's neurologist walked into the room. Illingworth requested the referral from her son's pediatrician based on instinct. George took ...

N.J. charity with Staten Island ties that was founded for teen with rare disease returns to NYC Marathon

“Kids with Duchenne muscular dystrophy, mostly boys, lose the ability to walk in their early teens,” Raffone said. “They lose ...

Unique bone disease doesn't stop 10-year-old Kid Captain from inspiring others

This week's Kid Captain for when the Hawkeyes take on the Ducks is 10-year-old Harper Atkinson of Wapello.Atkinson has a rare ...