A Personal Message from Our CEO

At Ultragenyx, our focus is developing medicines for people who live with rare and ultra-rare diseases.

We achieved a critical milestone in this mission when we received approval from the U.S. Food and Drug Administration for our first medicine in 2017. Our ongoing commitment is to help every patient who could benefit from our medicines to get access to treatment.

It takes more than scientific knowledge and research to develop medications. Your involvement is critical, because no one understands the impact that rare diseases have more than the people who live with them every day. Our advocacy team has the important role of partnering with patient groups to help us understand how the diseases affect your lives, so that we can develop programs that meet your needs. That two-way conversation is critical for us to find treatments where none currently exist.

I am a physician by training and have worked with families touched by rare diseases for more than two decades. I’ve been fortunate to be present when the first patients were successfully treated with new medications for conditions that previously had no therapies. Those moments have stayed with me, and our goal at Ultragenyx is to create those moments for more patients with rare diseases.

It is our privilege and responsibility to work with you and for you.


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Emil D. Kakkis, M.D., Ph.D.
Chief Executive Officer and President
Ultragenyx Pharmaceutical Inc.