Overview
Urea Cycle Disorder is treated with 11 medications in our database, including sodium phenylbutyrate, Sodium phenylacetate and Sodium benzoate, Carbaglu, SODIUM PHENYLACETATE AND SODIUM BENZOATE, Orapaz, and 6 more. 11 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Horizon, Bausch Health, Recordati, Horizon No punctuation., Amgen and others. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Urea Cycle Disorder treatment below.
Variable
Can be inherited in different ways depending on the underlying gene
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
4 eventsChildren's National Research Institute
University Children's Hospital, Zurich — NA
Children's National Research Institute
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
6 availableSodium Phenylbutyrate
indicated in all patients with neonatal-onset deficiency (complete enzymatic deficiency, presenting within the first 28 days of life)
Ravicti
RAVICTI is indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supp…
RAVICTI is indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
Glycerol Phenylbutyrate
indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation…
indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone
Orapaz
Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo
BUPHENYL, Buphenyl
Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo
Carbaglu
* Patient Copay Amount: $0 copay * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Must be a qualified patient with commercial insurance. Patient Assistance Progr
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford 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
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
2 resources3 travel grants are also available for Urea Cycle Disorder patients — see Travel Grants below ↓
Travel Grants
3 grantsRavicti Patient Access Liaisons (PALs) Travel Assistance
HealthWell Foundation Urea Cycle Disorders Fund
HealthWell Foundation
Applicants must be receiving treatment for Urea Cycle Disorders within the United States and possess health insurance that covers their medication. Eligibility also requires a household income at or below 500% of the Federal Poverty Level, adjusted for household size and cost of living.
Urea Cycle Disorders Financial Assistance Program
Applicants must be U.S. citizens or permanent residents diagnosed with a urea cycle disorder and prescribed an FDA-approved treatment with existing prescription coverage. Eligibility also requires meeting financial criteria based on household income and size to receive assistance with treatment-related costs and travel expenses.
Community
No community posts yet. Be the first to share your experience with Urea Cycle Disorder.
Start the conversation →Latest news about Urea Cycle Disorder
Disease timeline:
New recruiting trial: Ureagenesis Analysis in Healthy Subjects and in Urea Cycle Disorder Patients
A new clinical trial is recruiting patients for Urea Cycle Disorder
New recruiting trial: Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders
A new clinical trial is recruiting patients for Urea Cycle Disorder
New recruiting trial: Orphan Europe Carbaglu® Surveillance Protocol
A new clinical trial is recruiting patients for Urea Cycle Disorder
Caregiver 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 Urea Cycle Disorder
What is Urea Cycle Disorder?
Urea Cycle Disorder is treated with 11 medications in our database, including sodium phenylbutyrate, Sodium phenylacetate and Sodium benzoate, Carbaglu, SODIUM PHENYLACETATE AND SODIUM BENZOATE, Orapaz, and 6 more. 11 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Horizon, Bausch Health, Recordati, Horizon No punctuation., Amgen and others. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Urea Cycle Disorder treatment below.
Are there clinical trials for Urea Cycle Disorder?
Yes — 3 recruiting clinical trials are currently listed for Urea Cycle Disorder on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Urea Cycle Disorder?
17 specialists and care centers treating Urea Cycle Disorder are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Urea Cycle Disorder?
8 FDA-approved treatments and 2 patient support programs are currently tracked on UniteRare for Urea Cycle Disorder. See the treatments and support programs sections for copay assistance, eligibility, and contact details.