In the company of
friends, fighters, and the forever fearless.
We are inspired and motivated by the patients, families, and care partners we serve. We strive to develop treatments, resources, and educational materials that live up to our promise and your standards and empower patients to advocate on behalf of themselves and their rare communities.
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.
Emil D. Kakkis, M.D., Ph.D.
Chief Executive Officer and President
Ultragenyx Pharmaceutical Inc.
Role of Patient Advocacy
We support the vital work of patient organizations.
We educate by sharing information about diseases and their impact
We listen to the needs and views of patient communities to guide our work.
We partner with patient organizations to support patients and caregivers.
Meet the Advocacy Team
The Ultragenyx advocacy team works with patient advocacy groups to understand and represent the views of those living with rare diseases. They strive to provide valuable information, educational materials and resources to the communities we serve. Your input also helps guide our clinical development programs.
Together we share a common commitment of improving the lives of everyone affected by rare diseases.
If you’d like to share your story or learn more about advocacy at Ultragenyx, email [email protected]