Recent Posts
Travis’s Story of Wilson Disease
Rare and Rural By Rhonda Rowland, WDA President We all love a small town. Images come to mind of holiday [...]
Newborn Screening for Wilson Disease
A 25-year dream coming close to reality By Alice Williams, WDA Communications Director Since the 1960s, the United States has [...]
Can Copper be Absorbed Through the Skin?
By Edward Tabor, MD, WDA Board Member The main source of copper for humans is food, and Wilson disease patients [...]
They Said “Yes” to Gene Therapy
By Rhonda Rowland, WDA PresidentWe continue the story of two trailblazers who are among the first Wilson disease (WD) patients [...]
Meet the Inaugural Gene Therapy Candidates
By Rhonda Rowland, WDA President The two gene therapy clinical trials for Wilson disease (WD) launched by Ultragenyx and [...]
The 100th Registry Participant
By Rhonda Rowland, WDA President I’m study participant number 30 in the Wilson Disease (WD) Patient Registry. That’s not exciting. [...]
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.
