Ultragenyx Patient Advocacy

Rare and UltraRare Disease

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Regina, an older woman with brown hair, smiles at the camera

Regina

#RareStrength in Care Partnering

Creatine Transporter Deficiency (CTD)

“It takes a village to raise a kid, and in my experience with rare disease, it’s actually taken several villages.”

Meet Regina – mother, grandmother, and care partner to her daughter, Chelsi, and her grandson, Caiden, who both live with creatine transporter deficiency (CTD).

We recognize all those who care for someone rare. You are an essential part of the rare community.

Creatine Transporter Deficiency (CTD)

Learn more about creatine transporter deficiency, accessing related resources, and finding opportunities to participate in research for CTD.

Learn More about CTD

CTD Patient Advocacy Groups*

Learn about organizations from around the world who specialize in advocacy, support, education, and more.

*These organizations are an incomplete listing of rare disease advocacy groups 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. If you would like to have your group added to the list, please contact [email protected].

Rare Experiences

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Gabrielle

Angelman syndrome (AS)

“Angelman syndrome (AS) is part of your child, but it does not define who they are.”

Read More about Gabrielle's story

Juliet

Osteogenesis Imperfecta (OI)

“Your life does not stop after a diagnosis”

Read More about Juliet's story

Cynthia

Mucopolysaccharidosis Type VII (MPS VII)

“We would like Cynthia to know that she is not the only one with a rare disease”

Read More about Cynthia's story

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