Recent Posts
Celebrating Carol Terry: A Life of Resilience, Leadership, and Impact
Date: June 23, 2026 This June, we celebrate the 80th birthday of Carol Terry — co-founder of the Wilson [...]
My Experience Being Pregnant With Wilson Disease
By Carly Albinder, LCSW, WDA Patient Education and Support Director When I started thinking about getting pregnant, I knew [...]
You Are Not Alone: Kelsey’s Journey With Wilson Disease
By Kelsey Pusillo, WD patient and WDA Patient Ambassador Coordinator Early Symptoms and a Life-Changing Diagnosis During my high [...]
EL-PFDD Meeting for Wilson Disease: Patients and Caregivers Make Their Voices Heard
By Edward Tabor, MD, and Sarah Wicks, JD, MPH The WDA organized and led a daylong “Externally-Led Patient-Focused Drug [...]
Zinc and Me
By Rhonda Rowland, WDA President After 20 years of taking over-the-counter (OTC) zinc to treat my Wilson disease (WD), [...]
How One Family’s Wilson Disease Journey Led to a Transformative Legacy
By Rhonda Rowland, WDA President This past spring, I received a phone call that felt like being told we'd [...]
Ultragenyx’s Wilson Disease Gene Therapy Program Update
On behalf of the Ultragenyx Wilson disease study team, we would like to share a recent update on our CYPRUS2+ study.
Ultragenyx has changed Stage 1 of our CYPRUS2+ study to an open-label, single arm design. This means that all patients enrolled in the study will receive UX701, an investigational gene therapy for the treatment of Wilson disease. Stage 1 of the study no longer utilizes placebo, which will allow us to better understand the biological response to UX701 in real-time, as well as optimize the safe reduction of current treatment for WD.
The changes to the CYPRUS2+ protocol for Stage 1 may take place at different times depending on site locations. For sites located outside of the U.S., they must await approval by their country’s regulatory agency. Whether or when our investigational gene therapy will be approved by regulatory agencies as a treatment for Wilson disease is not known. The results from additional studies are needed to decide whether this treatment is safe and effective.
You are invited to email PatientAdvocacy@Ultragenyx.com for more information or read the full update located at: https://clinicaltrials.gov/ct2/show/NCT04884815?term=ux701&draw=2&rank=1.
