For Avery and Addison, we go beyond every day

Advancing medicines for rare diseases

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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

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

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Pipeline

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

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June 12, 2024

Ultragenyx Announces Proposed Public Offering of Common Stock and Pre-Funded Warrants

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June 12, 2024

Ultragenyx Announces Plans to File for Accelerated Approval of UX111 for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)

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June 11, 2024

Ultragenyx and Mereo BioPharma Announce New Phase 2 Data from Phase 2/3 Orbit Study Demonstrating Sustained Reductions in Fracture Rates Following Treatment with Setrusumab (UX143) in Patients with Osteogenesis Imperfecta (OI)

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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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