A research team has shown that that combining pembrolizumab, an immunotherapy drug, with standard chemotherapy can improve treatment outcomes for patients with small cell bladder cancer and small cell ...

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

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

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

Learn why it's making a comeback, its symptoms and what you can do about this disease caused by a vitamin C deficiency.

With the approval of MIPLYFFA, Zevra received a rare pediatric disease Priority Review Voucher (PRV) which the Company could monetize to help fund its growth. As of Oct. 31, Zevra has received 90 ...

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

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

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

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

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

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