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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Careers

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Latest news

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February 15, 2024

Ultragenyx Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Update

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February 08, 2024

Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2023 Financial Results and Corporate Update

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February 06, 2024

Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)

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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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