Acrofacial dysostosis

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ORPHA:364574
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3Specialists8Treatment centers

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Overview

Acrofacial dysostosis is a group of rare genetic conditions that affect the development of the face and limbs, particularly the hands and feet. The name comes from 'acro' meaning the extremities (hands and feet), 'facial' referring to the face, and 'dysostosis' meaning abnormal bone formation. People with this condition are typically born with underdeveloped cheekbones, a small jaw (micrognathia), downward-slanting eyes, and abnormalities of the outer ears. The limbs may show missing or underdeveloped fingers or toes, shortened forearms, and other bone abnormalities in the hands and feet. There are several subtypes of acrofacial dysostosis, including Nager syndrome and Miller syndrome, each caused by different genes and with slightly different features. The severity of symptoms can vary widely, even among family members with the same condition. Some individuals may have mild facial differences and minor limb involvement, while others may have more significant challenges including hearing loss, breathing difficulties due to a small airway, and feeding problems in infancy. Cleft palate is also common in many subtypes. Treatment is supportive and tailored to each person's needs. It may include surgery to correct facial or limb abnormalities, hearing aids, speech therapy, and specialized dental care. A team of specialists working together provides the best outcomes for people living with acrofacial dysostosis.

Key symptoms:

Underdeveloped or flat cheekbonesSmall or receding lower jawDownward-slanting eyesAbnormally shaped or small earsHearing lossCleft palate or high-arched palateMissing or shortened thumbsUnderdeveloped or missing fingers or toesShortened forearmsBreathing difficulties due to small airwayFeeding difficulties in infancyEyelid abnormalities such as colobomaSpeech difficultiesDental abnormalitiesAbsent or underdeveloped radius bone in the forearm

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Acrofacial dysostosis.

View clinical trials →

No actively recruiting trials found for Acrofacial dysostosis at this time.

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Specialists

3 foundView all specialists →
JP
Joy C MacDermid, PhD
Specialist
PI on 2 active trials
MP
Mark Sun, PhD
COON RAPIDS, MN
Specialist
PI on 1 active trial

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 Acrofacial dysostosis.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Acrofacial dysostosis

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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.What specific subtype of acrofacial dysostosis does my child have, and what gene is involved?,What surgeries might be needed, and when is the best timing for each?,How should we monitor and manage hearing loss?,What therapies (speech, occupational, feeding) should we start, and how soon?,Are there any breathing or airway concerns we need to watch for?,What is the expected long-term outlook for my child's development and independence?,Should other family members be tested for the genetic change?

Common questions about Acrofacial dysostosis

What is Acrofacial dysostosis?

Acrofacial dysostosis is a group of rare genetic conditions that affect the development of the face and limbs, particularly the hands and feet. The name comes from 'acro' meaning the extremities (hands and feet), 'facial' referring to the face, and 'dysostosis' meaning abnormal bone formation. People with this condition are typically born with underdeveloped cheekbones, a small jaw (micrognathia), downward-slanting eyes, and abnormalities of the outer ears. The limbs may show missing or underdeveloped fingers or toes, shortened forearms, and other bone abnormalities in the hands and feet. Ther

At what age does Acrofacial dysostosis typically begin?

Typical onset of Acrofacial dysostosis is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Acrofacial dysostosis?

3 specialists and care centers treating Acrofacial dysostosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.