Wilson Disease Blog
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 Vivet Therapeutics are underway! Here, we introduce the WD community to two people who are trailblazers in these studies that will show us if gene therapy works as a treatment [...]
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. However, if you’re participant number 100, you represent a milestone. And, that is exciting. Especially for those who’ve put in countless hours and tremendous effort to recruit patients, test them [...]




