Ultragenyx

Performance

About Ultragenyx

Ultragenyx is a biopharmaceutical company focused on developing first-ever approved treatments for rare and ultrarare genetic diseases and advancing therapies to move the rare disease community forward. The company operates with a global pipeline across multiple indications, including glycogen storage disease type Ia, OTC deficiency, Wilson disease, osteogenesis imperfecta, Angelman syndrome, MPS IIIA, and CDD, among others. Its research emphasizes novel modalities and comprehensive development and commercialization strategies to deliver transformative medicines for patients with limited or no existing options.