Zevra Therapeutics Inc. (ZVRA) said that the U.S. Food and Drug Administration approved MIPLYFFA (MY-PLY-FAH) (arimoclomol) capsules ...

Recent health developments include the EU backing Novo Nordisk's Wegovy for obesity-related heart conditions, FDA approvals ...

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

After participating in a 45-minute workout this week, 12 Tufts University men's lacrosse players have been diagnosed with a rare muscle injury known ...

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

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

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

Investigators developed the largest collection of sarcoma patient-derived organoids to date that can help improve the understanding of the disease and better identify therapies that are most likely to ...

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

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

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

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