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Blog2025-03-12T13:17:41+00:00

Wilson Disease Blog

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 to volunteer for one of the two gene therapy clinical trials now underway for our rare genetic liver disorder.  Emily signed up for the Ultragenyx trial and Warren volunteered for [...]

By |August 28, 2023|Categories: Blog Stories|0 Comments

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 [...]

By |March 27, 2023|Categories: Blog Stories|0 Comments

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 [...]

By |March 27, 2023|Categories: Blog Stories|0 Comments

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