Recent News and Updates

1103, 2022

Ultragenyx’s Wilson Disease Gene Therapy Program Update

March 11, 2022|

CYPRUS2+ is a randomized, double blind, placebo controlled, seamless Phase 1/2/3 study to assess safety, tolerability, and efficacy of a one-time intravenous (IV) infusion of UX701, an investigational AAV-Mediated Gene Therapy, for the treatment of Wilson Disease. Enrollment will include adults living with Wilson Disease currently being well managed on standard of care.

1003, 2022


March 10, 2022|

#GATEWAY is a Vivet Therapeutics clinical trial for #WilsonDisease, taking place at several clinical sites across the United States and Europe. Learn more about this currently recruiting clinical trial and our #Gene Therapy, VTX-801: [...]

2911, 2021

Wilson Disease Patient Registry Study

November 29, 2021|

The Wilson Disease Association (WDA) is not aware of any other multicenter, multinational Wilson Disease registry with prospective data collection in the world. The results of this research will benefit current and future WD patients [...]

1108, 2021

The Wilson Disease copper balance study

August 11, 2021|

The aim of this clinical study in people with Wilson Disease is to find out whether Alexion’s investigational product is able to help restore copper balance by promoting the removal of more copper from the [...]

1008, 2021

Ultragenyx Research Opportunities for Patients

August 10, 2021|

Multiple Ways to Participate in Wilson Disease Research Studies Who We Are Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company working to develop new products for the treatment of rare and ultra-rare diseases. We are committed [...]


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