Overview
Wilson Disease is treated with 1 medication in our database, including Depen. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Viatris. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Wilson Disease treatment below.
Also known as:
Clinical phenotype terms— hover any for plain English:
Autosomal recessive
Passed on when both parents carry the same gene change; often skips generations
Childhood to adulthood
Can begin any time from childhood through adulthood
FDA & Trial Timeline
10 eventsOrphalan — PHASE3
Fondation Ophtalmologique Adolphe de Rothschild
Yale University — NA
DSciLab Co., Ltd. — EARLY_PHASE1
Prime Medicine, Inc.
Assiut University
Centre Hospitalier Universitaire de Saint Etienne — NA
GeneCradle Inc — PHASE1, PHASE2
Thomas Damgaard Sandahl
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availablePenicillamine
indicated in the treatment of Wilson's disease
Galzin
For maintenance treatment of patients with Wilson's disease who have been initially treated with a chelating agent.
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersYale School of Medicine
📍 New Haven, Connecticut
👤 Sara Pai
Stanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
5 resourcesPAN Foundation — Wilson disease
PAN Foundation
Wilson disease
NORD Patient Assistance — Wilson disease
NORD Patient Assistance
Wilson disease
The Assistance Fund — Wilson disease
The Assistance Fund
Wilson disease
2 travel grants are also available for Wilson disease patients — see Travel Grants below ↓
Travel Grants
2 grantsWilson Disease Association Patient Assistance Program
Wilson Disease Association
Medically stable patients with rare diseases requiring travel over long distances for specialized care are eligible for free air and ground transportation. Services are provided at no cost to the patient and family for as long and as often as they need to fly.
Community
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1 articlesCaregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Wilson disease
What is Wilson disease?
Wilson Disease is treated with 1 medication in our database, including Depen. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Viatris. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Wilson Disease treatment below.
How is Wilson disease inherited?
Wilson disease follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Wilson disease typically begin?
Typical onset of Wilson disease is childhood to adulthood. Age of onset can vary across affected individuals.
Are there clinical trials for Wilson disease?
Yes — 13 recruiting clinical trials are currently listed for Wilson disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Wilson disease?
18 specialists and care centers treating Wilson disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Wilson disease?
1 FDA-approved treatment and 5 patient support programs are currently tracked on UniteRare for Wilson disease. See the treatments and support programs sections for copay assistance, eligibility, and contact details.