For families affected by this rare, fatal disease, the journey is grueling — not only because of the diagnosis itself but due ...

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 ...

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

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

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 ...

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

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

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

The U.K. drugmaker also announced plans to drop another $2 billion on expanding its R&D and manufacturing footprint in the ...