For Patients

Our Commitment to Patients

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company working to develop novel products for the treatment of rare and ultra-rare diseases.

Our focus is on improving the lives of patients who have no approved therapies for their diseases. Everyone in our company shares the same goal: delivering safe and effective therapies as quickly as possible to the people who need them.

We are passionate about educating and supporting patients, families and caregivers affected by rare and ultra-rare diseases. Visit our patient advocacy website for valuable resources, hear from people living with rare diseases, and learn more about our commitment to the rare disease patient community.

About Clinical Studies for Rare Diseases

The development of an investigational treatment is divided into steps called ‘phases’. The investigational treatment generally passes through each phase successfully before it can continue on to the next.

3-50 people 10-60 people 12-150 people
Usually healthy volunteers but can be people with the disease People with the disease People with the disease
Safety and how the body breaks down the study treatment

Safety and effectiveness (the ability of a study treatment to produce a beneficial change)

Long-term safety and effectiveness compared to current standard of care or a placebo (a sugar pill)

Once the studies are complete, the study information along with other information on the drug is submitted to and reviewed by a government agency (e.g., the United States Food and Drug Administration, or FDA). If the data shows the study treatment is safe and effective, it may be approved for use by prescription.

Ultragenyx is evaluating investigational treatments in clinical studies in a number of rare diseases. 

Rare Disease Organizations

Ultragenyx partners with several organizations across all rare diseases. These groups represent individual rare disease advocacy organizations to government. They contribute to research and fundraising efforts.

Global Genes


EveryLife Foundation  


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