Overview
Alagille Syndrome is treated with 2 medications in our database, including CTEXLI, Livmarli. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Mirum Pharmaceuticals, Mirum. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Alagille Syndrome treatment below.
Clinical phenotype terms— hover any for plain English:
Autosomal dominant
Passed on from just one parent; each child has about a 50% chance of inheriting it
Neonatal
Begins at or shortly after birth (first 4 weeks)
FDA & Trial Timeline
8 eventsChildren's Hospital of Eastern Ontario
Digestive Care, Inc. — PHASE2
Takeda
University Hospital, Toulouse
Mirum Pharmaceuticals, Inc. — PHASE4
Ipsen
Mirum Pharmaceuticals, Inc.
Albireo, an Ipsen Company — PHASE3
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availableCTEXLI
* Patient Copay Amount: As little as $0 per fill * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: For the Savings Program, patients must have commercial insuranc
Livmarli
* Patient Copay Amount: $10 or less per fill * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Must be a US resident and enrolled in the Mirum Access Plus (MAP) p
Treatment Centers
8 centersBoston Children's Hospital
📍 Boston, Massachusetts
Stanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🔬 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
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🏥 NORDBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
Travel Grants
1 grantsHealthWell Foundation Financial Assistance Programs
HealthWell Foundation
The HealthWell Foundation provides financial assistance to adults and children to cover out-of-pocket healthcare costs like prescription copayments, deductibles, and premiums. Eligibility is based on financial need relative to the Federal Poverty Level and having health insurance that is insufficient to cover treatment costs.
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 Alagille syndrome
What is Alagille syndrome?
Alagille Syndrome is treated with 2 medications in our database, including CTEXLI, Livmarli. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Mirum Pharmaceuticals, Mirum. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Alagille Syndrome treatment below.
How is Alagille syndrome inherited?
Alagille syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Alagille syndrome typically begin?
Typical onset of Alagille syndrome is neonatal. Age of onset can vary across affected individuals.
Are there clinical trials for Alagille syndrome?
Yes — 5 recruiting clinical trials are currently listed for Alagille syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Alagille syndrome?
16 specialists and care centers treating Alagille syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Alagille syndrome?
2 FDA-approved treatments and 1 patient support program are currently tracked on UniteRare for Alagille syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.