Leading the future of rare disease medicine for patients like Mason.

We use our experience, insight, and commitment to move the rare disease community forward.

Our goal is to provide medicines to those with limited options and to help patients face rare diseases head on, with courage and confidence.

Press Releases

Ultragenyx has acquired GeneTx to continue to advance development of GTX-102 for Angelman Syndrome. You can read more

here.

Pipeline

Our diverse portfolio of approved medicines and investigational therapies is advancing first- or best-in-class treatments for diseases that exhibit a clear biology for treatment and are severely underserved by current standard of care.

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Culture

We maintain an environment that puts people first. This includes patients, their families and caregivers, and our employees. We respect the responsibility that comes with developing medicines for rare diseases.

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Latest Announcements

Ultragenyx Reports Preliminary 2022 Revenue; Guidance for 2023 Revenue and Cash Usage; Pipeline Updates and 2023 Milestones

January 6, 2023

Ultragenyx to Present at the 41st Annual J.P. Morgan Healthcare Conference

January 4, 2023

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

December 21, 2022

COVID-19 Information

As the COVID-19 pandemic continues to evolve, we are focused on the health and safety of our community including patients, caregivers, healthcare providers, partners, and employees. For patients specifically, this means that we are committed to providing an uninterrupted supply of our medicines to the patients who rely on them.

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