Clinical Studies and Trials

Medicine is advanced through human volunteers.  Some studies test new therapies and others gather information.

Ultragenyx’s Wilson Disease Gene Therapy Program Update

March 11, 2022|0 Comments

Ultragenyx is pleased to announce that the first patient has been dosed in the CYPRUS2+ study, a one-time intravenous (IV) infusion of UX701, an investigational gene therapy for the treatment of Wilson disease. Ultragenyx is sponsoring this global study to determine if UX701 gene therapy is a safe and effective treatment for adults with Wilson disease. This adeno-associated virus (AAV) gene therapy has the potential to restore ATP7B function, which may improve copper metabolism and distribution.

Ultragenyx Update – Feb 17, 2022.

WDA Community Update – Jan 20, 2022.

The Wilson Disease copper balance study

August 11, 2021|0 Comments

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 body than is taken in through food and drink. This clinical study is currently recruiting, and if you would like to learn more, please contact Celerion Lincoln, Nebraska, at email: or call 866-445-7033. This clinical study is sponsored by Alexion Pharmaceuticals, Inc.

Ultragenyx Research Opportunities for Patients

August 10, 2021|0 Comments

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 to both developing new treatments and helping advance the understanding of rare diseases.

How You Can Help

We are currently developing an investigational gene therapy (UX701) for Wilson disease. We are committed to partnering with patients, caregivers, and family members.

There are multiple ways you can help. Please consider whether one or more studies below may be a good fit for you or your loved one. All studies below are institutional review board (IRB)-approved.



What’s involved


Interview Study Better understand the experience of people living with Wilson Disease


Questionnaires at home over 7 days Enrolling now
Odyssey Study Better understand how Wilson Disease appears and is managed medically


Get secure access to your medical records and contribute anonymized information from them for research Enrolling now
Antibody Study Better understand how many people have been exposed to a virus that is used in gene therapy


1 home visit with blood draw Enrolling soon
Clinical Survey Study Better understand Wilson disease by clarifying measures for clinical trials


1 clinic visit, 2 home visits over 35 days Enrolling now
Gene Therapy Phase 1/2/3 Study Evaluate an investigational gene therapy for the treatment of Wilson disease


One-time gene therapy infusion with multi-year follow-up Enrolling soon

If you are interested in learning more about how to get involved, please email


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Your support allows the WDA to maintain this website, produce educational materials, support research, and hold meetings for people living with WD, their families, and the healthcare community.

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Peer to Peer Fundraiser

Starting a fundraiser is easy to do, and it’s fun. We’ll even give you all the tools you’ll need to make a difference. With your help we can ensure critical steps of our 5 year plan get completed. This fundraiser requires no parties, walks or selling anything. You just send it out to people that you know. Contact Judi Keller at if you require further assistance with setting this up.
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We Are Wilsons

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We will be collecting patient stories and photographs to post on our website, Facebook and soon, Instagram. The Wilson Warrior stories have proven to be inspirational and we think we need a way to collect any story.

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Final Gift Plan

Consider WDA in a “Will” or Final Gift Plan
The Wilson Disease Association is 35 years old and is represented by board members from across the country and Canada. We have volunteers and partners globally and spearhead research that impacts our patients directly but affects a broader population beyond our patients. The long term importance of what the Wilson Disease Association does is limited only to the imagination because it affects generations of families around the world. Please consider the Wilson Disease Association in your will. Giving in this way is not restricted to the wealthy or a gender.

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Corporate Matches

Let us help you find employers and corporate donors to match your donation.
As a benefit to employees, many companies increase the impact of their employees’ gifts to WDA by providing matching contributions. Most corporations provide a 1:1 match; while some companies will double or even triple the matching amount to further encourage and leverage their employees’ philanthropy.

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Facebook Picture Post

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Help us spread the word by sharing a picture of yourself holding a sign with a message that we can post on Facebook and Instagram.

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Facebook Fundraiser

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We’ll help you get started and give you tips to reach your goal.

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Shop at AmazonSmile

You Shop. Amazon Gives.
Shop Online and Help the WDA. When you shop online at, designate the Wilson’s Disease Association as your charity of choice and a percentage of each sale (up to 12.5%) will be donated. Retailers include CD Now, J Crew, Avon, and many others. Log on for more information!

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