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Patient Focus

Many patients and families faced with the challenges of a rare or ultra-rare disease live with little hope, have limited or no treatment options, and face a shortened life-span. Every day, we strive to make a life-transforming impact on the lives of patients with severe and life-threatening rare disorders.

In the videos below, some of our patients share their experiences living with a rare disease. We hope that their stories inspire you as much as they inspire us.

Meet Joe
Diagnosed with PNH at 24 years old

 

 

Meet Erica
Diagnosed with aHUS at 22 years old

 

 

Meet Evie
Diagnosed with HPP at 2 weeks old