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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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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

Research

Grounded in science, guided by expertise

Developing life changing therapies for patients with serious rare and ultra-rare diseases

Science Card

Science

Learn how we develop therapies by modality.

Explore our science

Pipeline Card

Pipeline

See the progress we have made with our diverse portfolio of investigational therapies.

Explore our pipeline