Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome

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ORPHA:1812OMIM:225040Q87.8
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3Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome is an extremely rare genetic condition that affects multiple body systems at the same time. It is sometimes referred to by its Orphanet designation (ORPHA:1812) and falls under the broader group of ectodermal dysplasia syndromes combined with brain abnormalities. This syndrome involves problems with structures that develop from the ectoderm — the outer layer of the embryo — which gives rise to skin, hair, nails, teeth, and sweat glands. People with this condition typically have abnormalities in these areas, such as sparse hair, abnormal or missing teeth, and skin changes. In addition, the syndrome includes intellectual disability that can range from mild to severe, along with structural malformations of the brain and central nervous system. These brain abnormalities may include underdevelopment of certain brain regions and can contribute to seizures, developmental delays, and difficulties with movement or coordination. Because this condition is so rare, there is no specific cure or targeted therapy. Treatment focuses on managing individual symptoms. This may include special education services, physical and occupational therapy, dental care, seizure medications if needed, and dermatological support for skin and hair problems. A team of specialists working together is essential to provide the best possible care and quality of life.

Key symptoms:

Sparse or thin hairAbnormal or missing teethDry skin or reduced sweatingNail abnormalitiesIntellectual disabilityDelayed speech and language developmentDelayed motor milestones such as sitting and walkingSeizures or epilepsyBrain malformations visible on imagingUnusual facial featuresPoor muscle tone in infancyFeeding difficulties in early lifeShort stature or growth delaysVision or eye abnormalities

Clinical phenotype terms (39)— hover any for plain English
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 Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome.

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No actively recruiting trials found for Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome at this time.

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Specialists

3 foundView all specialists →
MP
Maria Lourdes Posadas Martinez, PhD
Specialist
PI on 1 active trial
MP
Marcelo Serra, PhD
Buenos Aires, Buenos Aires
Specialist

Rare Disease 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 Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome.

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Community

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Latest news about Ectodermal dysplasia-intellectual disability-central nervous system malformation 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.What specific brain abnormalities does my child have, and how might they affect development?,Is genetic testing available to confirm the diagnosis, and should other family members be tested?,What therapies (physical, occupational, speech) should we start, and how often?,How should we manage the risk of overheating if sweating is reduced?,Are there seizure medications that would be most appropriate, and what side effects should we watch for?,What dental treatments or prosthetics are recommended for missing or abnormal teeth?,Are there any clinical trials or research studies we could participate in?

Common questions about Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome

What is Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome?

Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome is an extremely rare genetic condition that affects multiple body systems at the same time. It is sometimes referred to by its Orphanet designation (ORPHA:1812) and falls under the broader group of ectodermal dysplasia syndromes combined with brain abnormalities. This syndrome involves problems with structures that develop from the ectoderm — the outer layer of the embryo — which gives rise to skin, hair, nails, teeth, and sweat glands. People with this condition typically have abnormalities in these are

How is Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome inherited?

Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome typically begin?

Typical onset of Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome?

3 specialists and care centers treating Ectodermal dysplasia-intellectual disability-central nervous system malformation syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.