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

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

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

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 ...

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

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 ...

The Siegel Rare Neuroimmune Association is a non-profit organization dedicated to the support of people with acute ...

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

AstraZeneca’s earnings were boosted by its oncology, cardiovascular, respiratory and immunology (R&I), and rare disease ...

The U.S. Food and Drug Administration on Tuesday declined full approval for Intercept Pharmaceuticals' liver disease drug, ...