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


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April 19, 2024

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

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April 15, 2024

Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102

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April 12, 2024

Ultragenyx Announces Update to Conference Call Timing to Discuss New Data from GTX-102 in Patients with Angelman Syndrome

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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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