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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.
Our Origin and Purpose
Ultragenyx’s origins are rooted in deep collaboration and engagement with the rare disease community, including families of patients with ultrarare genetic diseases. Dr. Emil Kakkis, our CEO and founder, has a legacy of creating research programs targeting conditions that impact a small number of people Dr. Kakkis created the EveryLife Foundation for Rare Diseases in 2009 to work on policy issues and advocate for patients who have rare diseases. Dr. Kakkis founded Ultragenyx on April 22, 2010 with the goal of creating a unique approach to drug development that 1) is responsive to severely underserved diseases with clear biology, 2) is opportunistic in applying different drug modalities to disease targets for rapid and nimble delivery of new medicines, and 3) drives close collaboration with the patient community to support optimal outcomes.
At Ultragenyx, we dedicate ourselves every day to the goal of serving the families in our rare disease communities by providing new, life changing treatments to patients. We also share a responsibility to our broader community of patients and their families and beyond, including our employees, customers, investors, collaborators, and our local neighborhoods. We have undertaken efforts that span: environmental sustainability; access to and pricing of our medicines; patient community engagement and support; inclusion, diversity and development of our team; and charitable contributions.