Recent News and Updates
Ultragenyx Wilson Disease Update April 2024
As Ultragenyx continues to advance its research for Wilson disease, please find a status update on the CYPRUS2+ study. The CYPRUS2+ study is designed with three stages. We are currently in Stage 1 where [...]
One Step Closer to a Newborn Screening Test for Wilson Disease
Key Proteo Submits De Novo Application to FDA for its First Newborn Screening Kit Potential to be the first IVD for the screening of Wilson Disease, Wiskott-Aldrich Syndrome, X-linked Agammaglobulinemia and Adenosine Deaminase Deficiency [...]
Ultragenyx Wilson Disease Gene Therapy Trial Update for WDA
As requested, we are providing an update on Stage 1 of our Cyprus2+ study, assessing safety and efficacy of UX701, an investigational gene therapy for the treatment of Wilson disease. The status update below [...]
Vivet Therapeutics’ Wilson Disease Gene Therapy Clinical Trial Update
On behalf of the Vivet Therapeutics Wilson disease study team, we are happy to share updates about the ongoing and enrolling GATEWAY clinical trial.GATEWAY is an international gene therapy clinical trial for people living with [...]
Wilson Disease Reviewed in Top Medical Journal
The Wilson Disease Association (WDA) is excited to share that the review article, “Current and Emerging Issues in Wilson’s Disease,” by Dr. Eve Roberts and Dr. Michael Schilsky is published in this week’s New [...]
They Said “Yes” to Gene Therapy
By Rhonda Rowland We continue the story of two trailblazers who are among the first Wilson disease (WD) patients to volunteer for one of the two gene therapy clinical trials now underway for our [...]
