Global developmental delay-osteopenia-ectodermal defect syndrome

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ORPHA:73223Q87.8
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Overview

Global developmental delay-osteopenia-ectodermal defect syndrome is a very rare genetic condition that affects multiple parts of the body at the same time. The name describes its three main areas of impact: the brain and nervous system (causing developmental delays), the bones (causing osteopenia, which means lower-than-normal bone density), and the ectodermal tissues — the skin, hair, nails, teeth, and sweat glands. Children with this condition typically show signs early in life, including delays in reaching milestones like sitting, walking, and talking. Their bones may be weaker than normal, raising the risk of fractures. Skin, hair, and nail changes are also common features. This syndrome is sometimes referred to by its Orphanet code ORPHA:73223 and falls under the broader category of multiple congenital anomaly syndromes. Because it is so rare, the full picture of how it affects people over a lifetime is still being studied. There is currently no cure, but supportive care — including physical therapy, occupational therapy, speech therapy, and bone health management — can help improve quality of life. A team of specialists working together is usually needed to manage all the different aspects of this condition.

Key symptoms:

Delayed development — slower than usual in learning to sit, walk, and talkIntellectual disability or learning difficultiesLow bone density (osteopenia), making bones more fragileIncreased risk of bone fracturesAbnormal skin texture or appearanceSparse or thin hairNail abnormalitiesDental problems, such as missing or abnormally shaped teethShort statureReduced sweating or abnormal sweat gland function

Clinical phenotype terms (39)— hover any for plain English
HypocalciuriaHP:0003127Hypoplastic sweat glandsHP:0007387Depigmentation/hyperpigmentation of skinHP:0007483Abnormality of hair textureHP:0010719Abnormality of dermal melanosomesHP:0011125Epidermal thickeningHP:0011368Orthokeratotic hyperkeratosisHP:0025080Abnormal temper tantrumsHP:0025160ImpulsivityHP:0100710
Inheritance

Autosomal recessive

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

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Global developmental delay-osteopenia-ectodermal defect syndrome.

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No actively recruiting trials found for Global developmental delay-osteopenia-ectodermal defect syndrome at this time.

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No specialists are currently listed for Global developmental delay-osteopenia-ectodermal defect syndrome.

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 Global developmental delay-osteopenia-ectodermal defect syndrome.

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Community

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Caregiver Resources

NORD Caregiver Resources

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Mental Health Support

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Family & Caregiver Grants

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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 genetic testing do you recommend to confirm the diagnosis, and how long will results take?,How often should my child have a bone density scan, and what treatments are available if the bones are very weak?,What early intervention therapies should we start right away, and how do we access them?,Are there any other specialists we should see as part of a care team?,What signs or symptoms should prompt us to go to the emergency room?,Is there a patient registry or research study we could join to help advance understanding of this condition?,What should we tell our child's school about their needs and how to keep them safe?

Common questions about Global developmental delay-osteopenia-ectodermal defect syndrome

What is Global developmental delay-osteopenia-ectodermal defect syndrome?

Global developmental delay-osteopenia-ectodermal defect syndrome is a very rare genetic condition that affects multiple parts of the body at the same time. The name describes its three main areas of impact: the brain and nervous system (causing developmental delays), the bones (causing osteopenia, which means lower-than-normal bone density), and the ectodermal tissues — the skin, hair, nails, teeth, and sweat glands. Children with this condition typically show signs early in life, including delays in reaching milestones like sitting, walking, and talking. Their bones may be weaker than normal,

How is Global developmental delay-osteopenia-ectodermal defect syndrome inherited?

Global developmental delay-osteopenia-ectodermal defect syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Global developmental delay-osteopenia-ectodermal defect syndrome typically begin?

Typical onset of Global developmental delay-osteopenia-ectodermal defect syndrome is infantile. Age of onset can vary across affected individuals.