Spondyloepiphyseal dysplasia, MacDermot type

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ORPHA:163668OMIM:184000Q77.7
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Overview

Spondyloepiphyseal dysplasia, MacDermot type (also called SED MacDermot type) is an extremely rare inherited bone disorder that belongs to a group of conditions known as skeletal dysplasias. These are conditions where the bones and cartilage do not develop properly. In this specific type, the spine (spondylo-) and the ends of the long bones near the joints (epiphyses) are primarily affected, leading to abnormal bone growth and short stature. People with this condition typically show signs in early childhood, including disproportionate short stature, a short trunk, and joint problems. The face may have distinctive features, and there can be abnormalities in the shape of the vertebrae (the bones of the spine) visible on X-rays. Some individuals may also experience joint stiffness or pain, waddling gait, and limitations in movement. Because this condition is so rare, with very few cases described in the medical literature, the full range of symptoms and the best approaches to treatment are not completely understood. Currently, there is no cure for this condition. Treatment is supportive and focuses on managing symptoms, which may include orthopedic interventions, physical therapy, and pain management. Regular monitoring by a team of specialists is important to address complications as they arise.

Also known as:

Key symptoms:

Short stature with a short trunkAbnormal shape of the spinal bones (platyspondyly)Underdeveloped ends of the long bones near jointsJoint stiffness or limited range of motionWaddling gait or difficulty walkingFacial features that may appear flattenedMild intellectual disability in some casesBarrel-shaped chestShort neckHip joint problemsBack pain or spinal curvature

Inheritance

X-linked recessive

Carried on the X chromosome; typically affects males more than females

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Spondyloepiphyseal dysplasia, MacDermot type.

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No actively recruiting trials found for Spondyloepiphyseal dysplasia, MacDermot type at this time.

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No specialists are currently listed for Spondyloepiphyseal dysplasia, MacDermot type.

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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 Spondyloepiphyseal dysplasia, MacDermot type.

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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 specific type of spondyloepiphyseal dysplasia does my child have, and how was this determined?,Is genetic testing available to confirm the diagnosis, and should other family members be tested?,What complications should I watch for, especially related to the spine?,How often should my child have X-rays or other imaging studies?,What physical activities are safe, and which should be avoided?,Are there any clinical trials or research studies we could participate in?,What support services are available for school and daily living?

Common questions about Spondyloepiphyseal dysplasia, MacDermot type

What is Spondyloepiphyseal dysplasia, MacDermot type?

Spondyloepiphyseal dysplasia, MacDermot type (also called SED MacDermot type) is an extremely rare inherited bone disorder that belongs to a group of conditions known as skeletal dysplasias. These are conditions where the bones and cartilage do not develop properly. In this specific type, the spine (spondylo-) and the ends of the long bones near the joints (epiphyses) are primarily affected, leading to abnormal bone growth and short stature. People with this condition typically show signs in early childhood, including disproportionate short stature, a short trunk, and joint problems. The face

How is Spondyloepiphyseal dysplasia, MacDermot type inherited?

Spondyloepiphyseal dysplasia, MacDermot type follows a x-linked recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Spondyloepiphyseal dysplasia, MacDermot type typically begin?

Typical onset of Spondyloepiphyseal dysplasia, MacDermot type is childhood. Age of onset can vary across affected individuals.