Atelosteogenesis type III

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ORPHA:56305OMIM:108721Q78.8
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Overview

Atelosteogenesis type III (AO3) is a very rare and severe bone development disorder that affects how the skeleton forms before birth. The name comes from Greek words meaning 'incomplete bone formation.' It is also sometimes called atelosteogenesis type 3. In this condition, many bones in the body do not develop properly, leading to very short limbs, an unusually small chest, and problems with the spine and joints. The bones may be partially missing or poorly formed, especially in the arms and legs. Babies born with AO3 typically have extremely short arms and legs, a narrow chest that makes breathing very difficult, and dislocated joints at the knees, hips, and elbows. The face may also look different, with a flat nasal bridge and a small jaw. Because the chest is so small, the lungs often cannot grow large enough to support breathing after birth, which makes this condition life-threatening in the newborn period. There is currently no cure for AO3. Treatment focuses on supporting breathing and managing complications. Because of the severe breathing problems caused by the small chest, most affected babies do not survive beyond the newborn period. Care is largely provided by a team of specialists focused on comfort and support for both the baby and the family.

Also known as:

Key symptoms:

Extremely short arms and legsVery narrow, small chestSevere breathing difficulties at birthDislocated joints, especially knees, hips, and elbowsUnderdeveloped or missing bones in the hands and feetFlat nasal bridgeSmall jaw (micrognathia)Abnormal spine development (scoliosis or other spinal problems)Short stature overallClubfoot or other foot deformitiesSoft, unstable joints

Clinical phenotype terms (27)— hover any for plain English
Abnormal humerus morphologyHP:0031095Short tubular bones of the handHP:0001248Patellar dislocationHP:0002999Coronal cleft vertebraeHP:0003417Knee dislocationHP:0004976Distal tapering femurHP:0006408Vertebral hypoplasiaHP:0008417Hand clenchingHP:0001188Fibular aplasiaHP:0002990Ulnar deviation of the wristHP:0003049Absent humerusHP:0003862Epiphyseal stippling of the humerusHP:0003902
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

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 Atelosteogenesis type III.

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No actively recruiting trials found for Atelosteogenesis type III at this time.

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No specialists are currently listed for Atelosteogenesis type III.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

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 Atelosteogenesis type III.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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Family & Caregiver Grants

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What does my baby's specific genetic test result mean for their prognosis?,What breathing support options are available, and what are the realistic outcomes of each?,What is the chance that a future pregnancy would be affected by AO3?,Can prenatal testing detect AO3 in a future pregnancy?,What palliative care and family support services are available to us?,Are there any clinical trials or research studies we should know about?,Can you connect us with other families who have been through this?

Common questions about Atelosteogenesis type III

What is Atelosteogenesis type III?

Atelosteogenesis type III (AO3) is a very rare and severe bone development disorder that affects how the skeleton forms before birth. The name comes from Greek words meaning 'incomplete bone formation.' It is also sometimes called atelosteogenesis type 3. In this condition, many bones in the body do not develop properly, leading to very short limbs, an unusually small chest, and problems with the spine and joints. The bones may be partially missing or poorly formed, especially in the arms and legs. Babies born with AO3 typically have extremely short arms and legs, a narrow chest that makes br

How is Atelosteogenesis type III inherited?

Atelosteogenesis type III follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Atelosteogenesis type III typically begin?

Typical onset of Atelosteogenesis type III is neonatal. Age of onset can vary across affected individuals.