Focused on Patient Communities

The patient advocacy team at Ultragenyx is passionate about educating and supporting you: patients, families and caregivers affected by rare and ultra-rare diseases. To find valuable educational resources, hear from others who live with rare diseases and more, please visit

What is the role of patient advocacy at Ultragenyx

The advocacy team understands the challenges facing many rare disease communities, including a lack of information and effective treatment options. We work with patient groups to provide communities with educational materials, and to ensure patients’ needs are shared within our company to guide our work.

Visit our website at to learn more about our commitment to you.

Your voice matters: We are listening

Ultragenyx Patient Advocacy


Raising a Child with LC-FAOD
“He (John) is focused on the present and determined not to let FAOD get in the way of life.”



Living with XLH
“My condition did not limit me prior to the diagnosis, and I was determined that it would never hold me back.”

Tara - Patient Brochure


Living with GNE Myopathy
“I’m motivated to help others get speedier testing and diagnosis by connecting them with the right support groups and resources.”



Raising a Child with Glut1 DS
“Talk to your doctor if something doesn’t seem right, and don’t shy away from difficult questions. Nobody knows your child better than you.”

Who is Ultragenyx?

Ultragenyx is a biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic conditions.


Patient Support Groups*

Ultragenyx works side by side with patient organizations that provide educational resources and support research to help people affected by
rare and serious disorders.

Rare Disease Groups
Global Genes
European Organization for Rare Disorders (EURORDIS)
National Organization for Rare Disorders (NORD)
Caregiver Action Network
EveryLife Foundation for Rare Diseases

Disease-Specific Groups
For information about disease-specific groups visit our Patient Support Resources page.

General Information about Clinical Trials

The Center for Information and Study on Clinical Research Participation (CISCRP)

*These organizations are an incomplete listing of rare disease support organizations and are not controlled by, endorsed by, or affiliated with Ultragenyx Pharmaceutical Inc. The list is meant for informational purposes only and is not intended to replace your healthcare professional’s medical advice. Ask your doctor or nurse any questions you may have about your disease or treatment plan.

MRCM-UGNX-00007 June 2016