Ultra-Committed
- - - To transform the lives of as many people living with rare diseases as possible, we dedicate ourselves every day to the development and delivery of treatments of rare diseases where none currently exist.
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  - Ultra-committed
Research
- - - We develop therapies for rare and ultrarare genetic diseases by exploring an array of drug modalities to select the best treatment strategy for each genetic disease we are studying.
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Medicines
Partnerships
Join Our Team
- - - We have built a talented team and a vibrant, people-first culture. We share a passion for helping those living with rare diseases.
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Newsroom
- - - Access news releases, presentations, and blog posts to follow the latest developments at Ultragenyx.
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Investors
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Press
releases

Feb 05, 2024

Ultragenyx Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome

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Jan 25, 2024

Ultragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease

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Jan 19, 2024

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

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Jan 07, 2024

Ultragenyx Reports Preliminary 2023 Revenue; Guidance for 2024 Revenue and Cash Usage; Pipeline Updates and 2024 Milestones

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Jan 04, 2024

Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

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Jan 03, 2024

Ultragenyx Announces Completion of Enrollment in Global Phase 1/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome

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Jan 02, 2024

Ultragenyx to Present at the 42nd Annual J.P. Morgan Healthcare Conference

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