Clinical Studies, Trials and Surveys

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

The Wilson Disease Association posts information here about clinical trials, studies and surveys that you can participate in. Please use this information to decide what is best for you.

Posts are screened for legitimacy.

Please remember, when you share your personal information or opinions in surveys, they benefit somebody and should be taken seriously. Use caution if asked to compete surveys by unknown parties that solicited you.

Posts are here to advance research in the treatment of Wilson disease. Many patients are interested in the same. The identities of participants posted here are kept anonymous from pharmaceutical companies.

Please review our terms of use.

Wilson Disease Patient Registry Study

Research Opportunity for Patients and Care Partners

June 20, 2022|0 Comments

Enviza, in collaboration with a major pharmaceutical company, is currently conducting an online research study to better appreciate the impact of Wilson disease on patients’ lives and to better understand how symptom and treatment changes over time impact patients’ lives. If you have been diagnosed with Wilson disease or if you are a care giver to someone diagnosed with Wilson disease, you may be qualified to participate.If you are interested in learning more or in participating in the research, click here.

Disclosure:  The Wilson Disease Association received compensation for reviewing this survey

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.

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.

Goal

Ages

What’s involved

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

18+

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

All

 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

18+

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

12+

 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

18+

 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 [email protected].

Donations

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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Membership

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.

Join Us!

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 [email protected] if you require further assistance with setting this up.
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We Are Wilsons

Share Your Story
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.

Learn More

Facebook Picture Post

Share a picture of yourself on Facebook
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.

Send a Pic

Facebook Fundraiser

Start your own fundraiser on Facebook
We’ll help you get started and give you tips to reach your goal.

Start Raising Money

Shop at AmazonSmile

You Shop. Amazon Gives.
Shop Online and Help the WDA. When you shop online at www.igive.com, 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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WILSON DISEASE ASSOCIATION

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