In the company of
parents, protectors, and proud partners in care.
Where are you in your
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.
Zoe’s StoryMucopolysaccharidosis type IIIA (also known as Sanfilippo syndrome type A)
Navigating through an Ultrarare Disease Diagnosis of Mucopolysaccharidosis type (MPS IIIA)
Michelle FynanOsteogenesis Imperfecta (OI)
“My Journey with Osteogenesis Imperfecta (OI) also known as Brittle Bone Disease”
AmandaWilson Disease (WD)
“Wilson disease affects many parts of my daily life because you literally have no idea what you are going to wake up to the next morning.”
Listen to a panel of incredible advocates moderated by The Two Disabled Dudes.
Upcoming Community EventsParticipate in Events Near You
The OIFE Annual General Meeting (AGM) will be held in Stockholm Sweden from June 10-11, 2023. All organizations are invited to attend and to speak at the annual general meeting, […]
The month of June is meaningful to the CDKL5 community. Every year in June, the CDKL5 community works to educate and raise awareness about the disorder.