Our Medicines

Developing innovative therapies for patients with serious rare and ultra-rare genetic diseases.

Our approach has led to the development of multiple first-ever rare disease treatments at a faster speed than the industry average.

ultragenyx medicine bottles

Crysvita®

Report negative side effects of prescription drugs to the FDA.

Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Dojolvi®

Report negative side effects of prescription drugs to the FDA.

Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Mepsevii®

Report negative side effects of prescription drugs to the FDA.

Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

ultragenyx medicine bottles

Our research and development team is hard at work developing novel therapies that we can bring to patients.

Read about our comprehensive pipeline of investigational treatments for patients in need