The Billings Livestock Commission hosted the sixth annual Calves to Cure to help raise money and awareness for Duchenne ...

The U.S. Food and Drug Administration has approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it ...

A rare-disease federal incentive program critical to biotech R&D is in jeopardy, writes Leslie Williams, president and CEO of ...

A 23-year-old Florida woman has been temporarily blinded and bruised after allegedly being required to receive multiple ...

Public health officials are investigating a cluster of nine confirmed cases of Legionnaires’ disease among people who ...

For Zevra Therapeutics and its rare neurodegenerative disease med arimoclomol, the second time’s the charm. | The ...

An Oscar-nominated actor recently spoke exclusively to PEOPLE about his adult son with a rare disease. It inspired a Rocky ...

Multiple system atrophy, or MSA, is a rare neurodegenerative disorder with symptoms akin to ALS or Parkinson’s disease in the ...

A St. George student's dreams came true Friday after hearing music he made after a rare disease took his sight and his voice.

The latest human EEE case in New York brings the national tally to at least 11 so far this year, according to an ABC News ...

Isabelle Cooper passed away on Thursday, a year after she was found to have the same type of genetic heart disorder as her ...

The FDA’s priority review voucher program for rare pediatric diseases is set to lapse on Sept. 30 without congressional intervention.