Dyssegmental dysplasia-glaucoma syndrome

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ORPHA:1804OMIM:601561
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Overview

Dyssegmental dysplasia-glaucoma syndrome is an extremely rare genetic condition that combines skeletal abnormalities (known as dyssegmental dysplasia) with eye problems, particularly glaucoma. Dyssegmental dysplasia refers to a group of bone growth disorders where the bones of the spine and limbs do not develop properly, leading to short stature, abnormal curvature of the spine, and shortened or bowed limbs. In this particular syndrome, these skeletal features occur alongside glaucoma, a condition where increased pressure inside the eye can damage the optic nerve and threaten vision. Patients with this syndrome may present at birth or in early infancy with noticeable shortening of the limbs, a narrow chest, and distinctive facial features. The glaucoma component can develop early in life and, if left untreated, may lead to significant vision loss. Other features may include joint stiffness, cleft palate, and hearing difficulties. Because this condition is so rare, there is no specific cure. Treatment focuses on managing individual symptoms. Orthopedic care may be needed for skeletal problems, while glaucoma is typically managed with eye drops, medications, or surgery to lower eye pressure. Early detection and a coordinated team of specialists are essential to optimize outcomes and quality of life for affected individuals.

Key symptoms:

Short statureShortened limbsAbnormal curvature of the spineNarrow chestGlaucoma (increased eye pressure)Joint stiffness or limited joint movementFlattened vertebrae in the spineDistinctive facial featuresCleft palateHearing problemsBowed bonesVision loss if glaucoma is untreatedBreathing difficulties due to small chest

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 Dyssegmental dysplasia-glaucoma syndrome.

View clinical trials →

No actively recruiting trials found for Dyssegmental dysplasia-glaucoma syndrome at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Dyssegmental dysplasia-glaucoma syndrome community →

No specialists are currently listed for Dyssegmental dysplasia-glaucoma 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 Dyssegmental dysplasia-glaucoma syndrome.

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Community

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Latest news about Dyssegmental dysplasia-glaucoma 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.How severe is my child's skeletal involvement, and what treatments are recommended?,How often should my child's eye pressure be checked for glaucoma?,Are there any breathing concerns related to the chest size, and what should I watch for?,What genetic testing should be done, and should other family members be tested?,What therapies (physical, occupational) would benefit my child?,Are there any clinical trials or research studies we could participate in?,What is the long-term outlook for my child's mobility and vision?

Common questions about Dyssegmental dysplasia-glaucoma syndrome

What is Dyssegmental dysplasia-glaucoma syndrome?

Dyssegmental dysplasia-glaucoma syndrome is an extremely rare genetic condition that combines skeletal abnormalities (known as dyssegmental dysplasia) with eye problems, particularly glaucoma. Dyssegmental dysplasia refers to a group of bone growth disorders where the bones of the spine and limbs do not develop properly, leading to short stature, abnormal curvature of the spine, and shortened or bowed limbs. In this particular syndrome, these skeletal features occur alongside glaucoma, a condition where increased pressure inside the eye can damage the optic nerve and threaten vision. Patients

How is Dyssegmental dysplasia-glaucoma syndrome inherited?

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

At what age does Dyssegmental dysplasia-glaucoma syndrome typically begin?

Typical onset of Dyssegmental dysplasia-glaucoma syndrome is neonatal. Age of onset can vary across affected individuals.