A toddler has died after being diagnosed with the same rare genetic disease that killed her three-year-old brother. Isabelle ...

Hiba Jelahej was standing in her kitchen in Chandler, Arizona, last summer when everything around her started spinning. She ...

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 ...

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 ...