In the company of
parents, protectors, and proud partners in care.
Where are you in your
rare journey?
The rare community is made up of millions of unique experiences. But shared experiences, common challenges, similar passions, and united voices tie everyone in it together. These Rare Journey Resources are designed to provide access to information for many of the common phases of the rare journey and empower you to reach out and get involved.
Understanding Drug Development
You have the power to improve and speed the drug development process. Visit Understanding Drug Development to learn more about how to get involved, and watch our video to see how Ultragenyx incorporates patients—including you— into our drug development.
In the company of rare.
Every experience with a rare disease or chronic illness is unique. The stories of those in the rare community strengthen, inspire, and drive us forward. You are the guardians, givers, greatest of groundbreakers, the advocates and altogether awe-inspiring. We’re proud to be in your company.
Cindy and Matthew
Osteogenesis Imperfecta (OI)“We refused to accept what we were being told, and I’m glad we did.”
Jonah
Glycogen Storage Disease Type Ia (GSDIa)“As I got older, I started learning how to listen to my body to know when I needed something.”
Tiffany and Wil
Duchenne Muscular Dystrophy (DMD)“In the beginning, we focused on what he was going to lose. Once we saw what he provided to those around him, Duchenne became just a part of who he is rather than his whole identity.”
Community Conversations
Listen to a panel of incredible advocates moderated by The Two Disabled Dudes.
Upcoming Community Events
Participate in Events Near YouAug 11
Join MitoAction and call host, FAOD Mom Stephanie Harry, on the second Thursday of each month for a support call for families affected by fatty acid oxidation disorders (FAOD). This […]
Sep 1
In September, organizations across the country raise awareness about the importance of screening newborns for potential illnesses and genetic disorders. Learn more
