Arthrogryposis-ectodermal dysplasia syndrome

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ORPHA:3200OMIM:601701Q68.8
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Overview

Arthrogryposis-ectodermal dysplasia syndrome is an extremely rare genetic condition that combines two major groups of problems: joint contractures (arthrogryposis) and abnormalities of structures that develop from the outer layer of the embryo, such as skin, hair, nails, teeth, and sweat glands (ectodermal dysplasia). Babies born with this condition typically have stiff, curved joints in their arms and legs that limit movement. They may also have thin or sparse hair, abnormal or missing teeth, dry skin, and reduced ability to sweat. Some affected individuals may also have other features such as growth delays or distinctive facial features. Because this syndrome is so rare, with only a handful of cases described in the medical literature, the full range of symptoms and their severity can vary between individuals. The condition is present from birth, and the joint problems are usually noticed right away. Treatment is supportive and focuses on managing the individual symptoms. Physical therapy and occupational therapy are important for improving joint mobility and function. Dental care is needed for tooth abnormalities, and skin care may be required for dryness. Orthopedic interventions, including bracing or surgery, may be considered for severe joint contractures. There is currently no cure for this condition, and management is lifelong and multidisciplinary.

Also known as:

Key symptoms:

Stiff or locked joints at birth (joint contractures)Limited range of motion in arms and legsThin or sparse hairAbnormal or missing teethDry skinReduced or absent sweatingBrittle or abnormal nailsShort stature or growth delaysDistinctive facial featuresDifficulty with fine motor skillsMuscle weakness or underdevelopment

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 Arthrogryposis-ectodermal dysplasia syndrome.

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No actively recruiting trials found for Arthrogryposis-ectodermal dysplasia syndrome at this time.

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No specialists are currently listed for Arthrogryposis-ectodermal dysplasia syndrome.

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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 Arthrogryposis-ectodermal dysplasia syndrome.

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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

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 is the specific genetic cause in our case, and should we pursue whole exome sequencing?,What therapies should we start right away to improve joint mobility?,How can we safely manage the risk of overheating if sweating is reduced?,What dental treatments will be needed as my child grows?,Are there any clinical trials or research studies we could participate in?,What is the chance of having another child with this condition?,What adaptive equipment or school accommodations should we consider?

Common questions about Arthrogryposis-ectodermal dysplasia syndrome

What is Arthrogryposis-ectodermal dysplasia syndrome?

Arthrogryposis-ectodermal dysplasia syndrome is an extremely rare genetic condition that combines two major groups of problems: joint contractures (arthrogryposis) and abnormalities of structures that develop from the outer layer of the embryo, such as skin, hair, nails, teeth, and sweat glands (ectodermal dysplasia). Babies born with this condition typically have stiff, curved joints in their arms and legs that limit movement. They may also have thin or sparse hair, abnormal or missing teeth, dry skin, and reduced ability to sweat. Some affected individuals may also have other features such a

How is Arthrogryposis-ectodermal dysplasia syndrome inherited?

Arthrogryposis-ectodermal dysplasia syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Arthrogryposis-ectodermal dysplasia syndrome typically begin?

Typical onset of Arthrogryposis-ectodermal dysplasia syndrome is neonatal. Age of onset can vary across affected individuals.