Overview
Homocystinuria is treated with 3 medications in our database, including Cystadane, EnBrace HR, Betaine. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Recordati, Jaymac. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Homocystinuria treatment below.
Autosomal recessive
Passed on when both parents carry the same gene change; often skips generations
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
8 eventsUniversity Children's Hospital, Zurich
Hamad Medical Corporation — NA
University of British Columbia — NA
Travere Therapeutics, Inc. — PHASE3
Travere Therapeutics, Inc. — PHASE3
Travere Therapeutics, Inc. — PHASE1, PHASE2
Travere Therapeutics, Inc.
Treatment of homocystinuria to decrease elevated homocysteine blood levels.
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
3 availableBetaine Anhydrous
indicated for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations
Cystadane
Treatment of homocystinuria to decrease elevated homocysteine blood levels.
EnBrace HR
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
Rare Disease Specialist
Rare Disease Specialist
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
1 resources1 travel grant are also available for Homocystinuria patients — see Travel Grants below ↓
Travel Grants
1 grantsTravere Therapeutics Patient Support Programs
Community
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Start the conversation →Latest news about Homocystinuria
Disease timeline:
New recruiting trial: Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
A new clinical trial is recruiting patients for Homocystinuria
New recruiting trial: Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency)
A new clinical trial is recruiting patients for Homocystinuria
New trial: Proof of Concept Creatine Supplementation for Homocystinuria Study
Phase NA trial recruiting. Creatine
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 Homocystinuria
What is Homocystinuria?
Homocystinuria is treated with 3 medications in our database, including Cystadane, EnBrace HR, Betaine. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Recordati, Jaymac. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Homocystinuria treatment below.
How is Homocystinuria inherited?
Homocystinuria follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Homocystinuria?
Yes — 6 recruiting clinical trials are currently listed for Homocystinuria on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Homocystinuria?
22 specialists and care centers treating Homocystinuria are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Homocystinuria?
3 FDA-approved treatments and 2 patient support programs are currently tracked on UniteRare for Homocystinuria. See the treatments and support programs sections for copay assistance, eligibility, and contact details.