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.

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.

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