The Wilson Disease Voice of the Patient Report captures the experiences, treatment challenges, and priorities shared by people living with Wilson disease, family members, and caregivers during the January 29, 2026, Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting.

Approximately 350 people joined the virtual meeting, including 40 representatives from the U.S. Food and Drug Administration. Community members also contributed through online polling, surveys, recorded testimony, and written comments.

The report is intended to help the FDA, researchers, clinicians, and drug developers better understand what matters most to the Wilson disease community and inform future treatment development and clinical trial design.

Participants described the wide-ranging hepatic, neurologic, and psychiatric effects of Wilson disease as well as the impact of symptoms and treatment on daily life. They also emphasized the need for therapies that slow or stop disease progression, improve neurologic symptoms, reduce treatment burden, and remain accessible and affordable.

Online Poll Results

The polling report provides a snapshot of participants’ experiences with symptoms, daily activities, current treatments, treatment drawbacks, and priorities for future therapies. The results are intended to complement the personal testimony shared during and after the meeting.

Submitted Comments and Survey Responses

This companion document includes comments submitted before, during, and after the meeting, along with responses from a community survey conducted in advance. It preserves additional patient and caregiver perspectives that may not appear in the main report.

Watch the January 29, 2026, EL-PFDD meeting that served as the foundation for the Voice of the Patient Report.

We are deeply grateful to the people living with Wilson disease, family members, and caregivers who shared their personal experiences for the EL-PFDD meeting. Their stories illustrate the many ways Wilson disease affects individuals and families.

Watch All Patient and Caregiver Stories on YouTube