Wilson disease Advocacy Hub | UniteRare

At a Glance

Live from database

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Regulatory Watchboard

10 events

Jun 2026Endocrine Dysfunction in Pediatric Wilson's Disease

Assiut University

TrialNOT YET RECRUITING

May 2026Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

Orphalan — PHASE3

TrialNOT YET RECRUITING

Feb 2026Description of Renal Involvement in Wilson's Disease

Fondation Ophtalmologique Adolphe de Rothschild

TrialNOT YET RECRUITING

Jan 2026Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study

Yale University — NA

TrialRECRUITING

Dec 2025A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)

DSciLab Co., Ltd. — EARLY_PHASE1

TrialRECRUITING

Dec 2025Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial

Prime Medicine, Inc.

TrialRECRUITING

Dec 2025Multifaceted Assessment of Patients With Wilson's Disease in a Low-Resource Setting in Upper Egypt: Service Integration, Psychosocial Burden, Dietary Practices, and the Geo-Spatial Disease Map

Assiut University

TrialNOT YET RECRUITING

Nov 2025Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity

Centre Hospitalier Universitaire de Saint Etienne — NA

TrialRECRUITING

Oct 2025Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)

GeneCradle Inc — PHASE1, PHASE2

TrialNOT YET RECRUITING

Jul 2025Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT

Thomas Damgaard Sandahl

TrialENROLLING BY INVITATION

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

7 programs

FINANCIAL LANDSCAPE SUMMARY

Total programs

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Foundation grants

Copay cards

Travel grants

Foundation Grants3

PAN Foundation — Wilson disease

PAN Foundation

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NORD Patient Assistance — Wilson disease

NORD Patient Assistance

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The Assistance Fund — Wilson disease

The Assistance Fund

OpenContact for detailsApply ↗

Copay Assistance1

Penicillamine

Bausch Health

OpenContact for detailsApply ↗

Patient Assistance Programs1

Galzin

Eton Pharmaceuticals

OpenContact for detailsApply ↗

Travel Grants2

RELIVE Organization Medication Assistance

RELIVE Organization

Contact for detailsApply ↗

Wilson Disease Association Patient Assistance Program

Wilson Disease Association

OpenContact for detailsApply ↗

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Approved Treatments

2 FDA-approved

Galzin

(Zinc acetate)Orphan drug

Eton Pharmaceuticals, Inc

Approved Jan 1997FDA label ↗

Penicillamine

(PENICILLAMINE)Orphan drugstandard

Endo USA, Inc.

FDA label ↗

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Clinical Trial Landscape

13 active trials

2N/A

8Unknown

2EARLY_PHASE1

1PHASE1, PHASE2

13Total recruiting

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Recent News & Research

1 article

ClinicalTrials.govMar 15, 2026

Wilson Disease Gene Therapy Enters Clinical Trials

First gene therapy trial for Wilson disease began enrolling, aiming to restore ATP7B protein expression.

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Specialist Network

Top 6 by expertise

Ultragenyx Medical Director

Ultragenyx Pharmaceuticals Inc.

📍 Little Rock, Arkansas

Score 234 trials

Michael L Schilsky, MD FAASLD

Yale University

📍 New Haven, Connecticut

Score 31 trial

Marcelo Serra, PhD

HIBA

📍 Buenos Aires, Buenos Aires

Score 31 trial

Liang Peng, Doctor, O.M.D, L. Ac

Third Affiliated Hospital, Sun Yat-Sen University

📍 HOUSTON, TX

Score 38 trials

Arndt Rolfs, Prof. Dr.

CENTOGENE GmbH Rostock

Score 24 trials

Aurélia POUJOIS, MD

Fondation A. de Rothschild

Score 22 trials1 pub

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Financial Assistance

Copay cards, PAPs, grants

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