Athabaskan brainstem dysgenesis syndrome

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ORPHA:69739OMIM:601536G96.8
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1Specialists8Treatment centers

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Overview

Athabaskan brainstem dysgenesis syndrome (ABDS) is a rare genetic condition that affects the development of the brainstem — the part of the brain that controls many basic body functions like breathing, eye movement, and swallowing. The syndrome is named after the Athabaskan-speaking Native American peoples of North America, where it was first identified and is most commonly found. It is sometimes also called Bosley-Salih-Alorainy syndrome (BSAS), and both names refer to the same condition caused by changes in the HOXA1 gene. Babies born with ABDS typically show signs very early in life. The brainstem does not form properly before birth, which leads to a range of serious problems. These include the inability to move the eyes normally, hearing loss, and difficulty breathing or swallowing. Some children also have heart defects and problems with the way blood vessels in the neck and chest are arranged. Intellectual disability and autism spectrum features have also been reported in some individuals. There is currently no cure for ABDS. Treatment focuses on managing symptoms and supporting the child's development. This may include breathing support, hearing aids or cochlear implants, heart surgery if needed, and therapies such as speech, occupational, and physical therapy. Early intervention and a team of specialists can make a meaningful difference in quality of life.

Also known as:

Key symptoms:

Inability to move the eyes side to side (horizontal gaze palsy)Hearing loss, often severe or profoundBreathing difficulties, especially in newbornsDifficulty swallowingFacial weakness or paralysisHeart defects present at birthAbnormal arrangement of large blood vessels in the chest and neckIntellectual disabilityAutism spectrum featuresAbsent or reduced reflexesAbnormal brainstem structure seen on brain scans

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Athabaskan brainstem dysgenesis syndrome.

View clinical trials →

No actively recruiting trials found for Athabaskan brainstem dysgenesis syndrome at this time.

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Search ClinicalTrials.gov ↗Join the Athabaskan brainstem dysgenesis syndrome community →

Specialists

1 foundView all specialists →

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Athabaskan brainstem dysgenesis syndrome.

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Community

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Latest news about Athabaskan brainstem dysgenesis syndrome

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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.Which specific mutation in the HOXA1 gene does my child have, and what does that mean for their prognosis?,Should other family members be tested to see if they are carriers?,What specialists should be part of my child's care team, and how often should we see each one?,Is my child a candidate for a cochlear implant, and when would be the best time to consider it?,What early intervention or therapy services should we start right away?,Are there any clinical trials or research studies we should know about?,What signs should prompt me to seek emergency care, and what should I do in a breathing emergency?

Common questions about Athabaskan brainstem dysgenesis syndrome

What is Athabaskan brainstem dysgenesis syndrome?

Athabaskan brainstem dysgenesis syndrome (ABDS) is a rare genetic condition that affects the development of the brainstem — the part of the brain that controls many basic body functions like breathing, eye movement, and swallowing. The syndrome is named after the Athabaskan-speaking Native American peoples of North America, where it was first identified and is most commonly found. It is sometimes also called Bosley-Salih-Alorainy syndrome (BSAS), and both names refer to the same condition caused by changes in the HOXA1 gene. Babies born with ABDS typically show signs very early in life. The b

How is Athabaskan brainstem dysgenesis syndrome inherited?

Athabaskan brainstem dysgenesis syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Athabaskan brainstem dysgenesis syndrome typically begin?

Typical onset of Athabaskan brainstem dysgenesis syndrome is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Athabaskan brainstem dysgenesis syndrome?

1 specialists and care centers treating Athabaskan brainstem dysgenesis syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.