California’s recent decision to create a Rare Disease Advisory Council is a milestone that brings new hope to people living ...

In Tulsa, mechanic Matt Parker rallied the community to support 21-year-old Corbin Norcom, who has a fatal disease, by ...

A new treatment that could prevent blindness in children with the CLN2 type Batten disease has been trialed by clinicians and ...

In the wake of an unfavorable advisory committee meeting in September, Intercept Pharmaceuticals’ bid to win full approval ...

Elraglusib has been granted rare pediatric disease designation from the FDA for the treatment of patients with Ewing sarcoma.

University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...

Researchers at Washington University have discovered a new genetic disorder affecting protein folding, offering potential new treatment avenues for rare brain conditions. When most people feeling ...

British drugmaker AstraZeneca increased its full-year sales outlook on booming demand for its cancer and rare-disease drugs, and said it plans to invest $3.5 billion in its U.S. business by the end of ...

Audrey Tyrrell, from Harwich, Essex, was diagnosed with Usher syndrome, a rare genetic disease that affects both hearing and ...

A practical calculator that predicts the risk of epilepsy after venous stroke is presented in a study from the University of Gothenburg and others.

Sessions that focused on diagnosis and acute treatment of rare neuroimmune disorders (RNDs). A panel of people with these ...

Ocaliva received the FDA's accelerated approval in 2016 to treat patients with primary biliary cholangitis, a rare disease ...