Overview
Biliary atresia and associated disorders (also called BA or biliary atresia spectrum) is a rare liver condition that mainly affects newborns and very young infants. In biliary atresia, the bile ducts — the small tubes that carry bile from the liver to the intestine — are blocked, scarred, or missing. Bile is a digestive fluid made by the liver. When it cannot drain properly, it builds up inside the liver and causes serious damage over time. This damage can lead to liver scarring (cirrhosis) and liver failure if not treated quickly. The most noticeable early signs are yellowing of the skin and eyes (jaundice) that does not go away after the first few weeks of life, pale or chalky-white stools, and dark yellow urine. Babies may also have a swollen belly and poor weight gain. Some children have other birth differences affecting the heart, spleen, or intestines — this is sometimes called biliary atresia splenic malformation (BASM) syndrome. Treatment must happen as early as possible. The first step is usually a surgery called the Kasai procedure (hepatoportoenterostomy), which tries to restore bile flow. Many children still need a liver transplant later in life. With early surgery and transplant when needed, many children can live into adulthood, though ongoing medical care is always required.
Key symptoms:
Yellowing of the skin and whites of the eyes (jaundice) lasting beyond 2 weeks of agePale, chalky, or clay-colored stoolsDark yellow or brown urineSwollen or enlarged bellyPoor weight gain or failure to thriveEnlarged liverEnlarged spleenItchy skinFatigue and low energyEasy bruising or bleedingFluid buildup in the belly (ascites) in advanced casesDelayed growth and development
Multifactorial
Caused by a mix of several genes and environmental factors
Neonatal
Begins at or shortly after birth (first 4 weeks)
FDA & Trial Timeline
2 eventsNational Liver Institute, Egypt — NA
Mirum Pharmaceuticals, Inc. — PHASE3
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Biliary atresia and associated disorders.
1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →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
Travel Grants
No travel grants are currently matched to Biliary atresia and associated disorders.
Community
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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.
Questions for your doctor
Bring these to your next appointment
- Q1.How quickly does my baby need the Kasai procedure, and what happens if we wait?,What are the chances the Kasai procedure will work well enough to delay or avoid a liver transplant?,What vitamins and nutritional supplements does my child need, and how do I give them?,When should we start thinking about a liver transplant evaluation?,Are there any clinical trials or new treatments we should know about?,What signs should make me call the hospital immediately?,How will this condition affect my child's growth, development, and school life long-term?
Common questions about Biliary atresia and associated disorders
What is Biliary atresia and associated disorders?
Biliary atresia and associated disorders (also called BA or biliary atresia spectrum) is a rare liver condition that mainly affects newborns and very young infants. In biliary atresia, the bile ducts — the small tubes that carry bile from the liver to the intestine — are blocked, scarred, or missing. Bile is a digestive fluid made by the liver. When it cannot drain properly, it builds up inside the liver and causes serious damage over time. This damage can lead to liver scarring (cirrhosis) and liver failure if not treated quickly. The most noticeable early signs are yellowing of the skin and
How is Biliary atresia and associated disorders inherited?
Biliary atresia and associated disorders follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Biliary atresia and associated disorders typically begin?
Typical onset of Biliary atresia and associated disorders is neonatal. Age of onset can vary across affected individuals.
Are there clinical trials for Biliary atresia and associated disorders?
Yes — 1 recruiting clinical trial is currently listed for Biliary atresia and associated disorders on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Biliary atresia and associated disorders?
3 specialists and care centers treating Biliary atresia and associated disorders are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.