Our Purpose

Leading the future of rare disease medicine

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Ultragenyx was founded to create a steady pipeline of new and effective therapies for patients with rare and ultra-rare diseases. It is our shared belief that we will achieve this by creating a people-first ethos that puts patient communities and our employees at the center of our approach and mission.


At Ultragenyx, our goal is to bring novel therapies to patients with serious rare and ultra-rare genetic diseases. We are targeting multiple therapeutic areas in which we have deep expertise: endocrine/bone diseases, metabolic diseases, and central nervous system (CNS)/muscle diseases.

Our team has a proven track record of identifying, developing, and launching medicines for rare diseases. Since our founding in 2010, we’ve launched three FDA-approved approved treatments in four indications, and developed a diverse portfolio of investigational therapies focused on modifying diseases. We put patients and their caregivers at the center of our work, involving them at all stages of therapeutic development. This provides us with an understanding of the impact a disease has on a patient – biologically, functionally, and emotionally – so we can address the entire person and not just their symptoms.

All of our efforts – from translational research to clinical development to patient access programs – are rooted in our commitment to provide patients and their families with courage and confidence today.

Learn more about how we support patients and their families

Grant Programs

We are committed to improving the lives of patients with rare and ultra-rare genetic diseases through educational initiatives, patient advocacy, research, and access to information. Our specific focus is on patient and professional organizations that provide vital support to the rare disease community.

Learn more about our grant programs

Our Ethics

We are committed to maintaining the highest standards of honest and ethical business conduct, and we make this commitment to the patients we treat, our business partners, our customers, our shareholders, our community, the government agencies that regulate our business, and ourselves.

View our Global Code of Conduct