For Mason, we go beyond every day

Advancing medicines for rare disease

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Leading with purpose. Every day.

“Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.”

Emil D. Kakkis, M.D., Ph.D.
Chief Executive Officer and President


Leadership Home

Our team of passionate biopharmaceutical leaders combines deep expertise in drug development and commercialization

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Pipeline Card

We have one of the largest and most diverse pipelines in rare disease

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June 05, 2023

Ultragenyx and Mereo BioPharma Announce Positive Data from the Ongoing Phase 2/3 Orbit Study of Setrusumab (UX143) in Osteogenesis Imperfecta (OI)

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May 24, 2023

Ultragenyx Issues 2022 Environmental, Social and Governance (ESG) Report

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May 19, 2023

Ultragenyx to Present at the 2023 Barclays - Gene Editing & Gene Therapy Summit

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Rare Perspective Home

Rare perspective

Insights and information on our approach to collaborating with rare disease communities, supporting our teammates at Ultragenyx, and developing new therapies for rare disease

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