Isabelle Cooper, 2 , died on Thursday September 19, just three years after her brother Alexander died of the same heart ...

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 ...

(RTTNews) - Zevra Therapeutics Inc. (ZVRA) said that the U.S. Food and Drug Administration approved MIPLYFFA (MY-PLY-FAH) (arimoclomol) capsules as an orally delivered treatment for Niemann-Pick ...

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.

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

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

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 ...

Parents pushed for years for treatments for the neuron disease Niemann-Pick type C. After setbacks, one drug now has FDA ...

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