(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.

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 ...

For the first time in a decade, health officials in New York confirmed a human case of a rare but serious mosquito-borne ...

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

First used in the 1940s to monitor for polio, wastewater surveillance proved such a powerful disease monitoring tool that the U.S. Centers for Disease Control and Prevention (CDC) established the ...

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 ...