NEJM Article

Rare Affair 2024

WD Low Cost Drugs

UNMASKING STRENGTH. UNLEASHING PROMISE.

The WDA aspires to unmask the challenges of Wilson disease and unleash the promise of a cure.

Wilson disease is an inherited disorder that causes too much copper to accumulate in the body.

Events

Upcoming events including the Annual Conference, local events, webinars, support group meetings and fundraisers.

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.

Clinical Trials and Studies

Find out about trials of new therapies and other studies now underway.

Gateway Trial is now Actively Enrolling at 3 US Clinical Sites

March 2022

The first gene therapy study of VTX-801 in adults with Wilson disease is open for enrollment.

How Mark Cuban helped a Cherry Hill business exec cut prices for the drug that keeps him alive

June 2023

For decades, the drug that keeps Drew Katz alive sold for less than $1 a pill.

Ultragenyx’s Wilson Disease Gene Therapy Program Update

Nov 2023

An update on Stage 1 of our Cyprus2+ study, assessing safety and efficacy of UX701, an investigational gene therapy for the treatment of Wilson disease.

Do I Have Wilson Disease?

Learn more about the signs and symptoms of Wilson disease.

Medical Professionals

Diagnosis, examinations, and treatment of Wilson disease.

Living With Wilson Disease

Learn more about treatments, patient assistance, and studies.

Registry for Patients with Wilson Disease

The registry is not only important to current patients but is important because it will impact patients for generations. Our researchers and our donors are creating a legacy. We have been very fortunate to meet the needs of our first two years but we cannot become complacent considering the amount needed to fund the remainder of the project.

WILSON DISEASE ASSOCIATION

Get involved!

There are so many ways to spread the word about Wilson disease and help friends and family affected.

Mission Statement

The WDA provides support and hope to people impacted by Wilson disease worldwide so that they may lead the best quality of life possible.