Recent News and Updates
Meet the Inaugural Gene Therapy Candidates
The two gene therapy clinical trials for Wilson disease (WD) launched by Ultragenyx and Vivet Therapeutics are underway! Here, we introduce the WD community to two people who are trailblazers in these studies that will [...]
A New Guidance on Wilson Disease
Diagnosis|Treatment|Monitoring By Rhonda Rowland This is a first for Wilson disease (WD). The new Practice Guidance on WD is a careful balance of combining limited scientific evidence with expert experience to create a tool for [...]
The 100th Registry Participant
By Rhonda Rowland I’m study participant number 30 in the Wilson Disease (WD) Patient Registry. That’s not exciting. However, if you’re participant number 100, you represent a milestone. And, that is exciting. Especially for those [...]
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 [...]
GATEWAY IS NOW ACTIVELY ENROLLING AT 3 US CLINICAL SITES!
It now has a dedicated website for the patient community and also a dedicated call center for US patients with a patient liaison you can speak with. website address is: https://www.gatewaytrialwilsondisease.com/ #GATEWAY is a Vivet Therapeutics [...]
Shipping Trientine to India through MAP International
The WDA is no longer able to ship Trientine to India through MAP International due to Indian rules. If you have any questions please contact: Dr Seema Alam Prof Pediatric Hepatology Department of Pediatric Hepatology [...]
