Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome

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ORPHA:2064OMIM:192800Q87.5
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Overview

Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome is an extremely rare genetic condition that combines abnormalities of the lower spine with drooping of the upper eyelids (called blepharoptosis or ptosis). In this syndrome, some of the bones in the lower back and sacral area (the lumbosacral vertebrae) are fused together at the back, meaning they are joined when they should normally be separate. At the same time, affected individuals have drooping eyelids, which can affect one or both eyes and may interfere with vision if severe. This condition falls under a group of disorders involving multiple birth defects affecting different body systems. Because the spine is involved, some people may experience stiffness or reduced flexibility in the lower back, and in some cases there may be associated neurological symptoms depending on how the spinal fusion affects nearby nerves. The eyelid drooping can range from mild to severe and may require surgical correction if it blocks the line of sight, especially in young children where it could affect visual development. Because this syndrome is so rare, there is very limited information about its full range of symptoms, natural history, and optimal treatment. Management is generally supportive and symptom-based, meaning doctors address each problem individually. Surgical options may be considered for the ptosis, and orthopedic care may be needed for spinal issues. Genetic counseling is recommended for affected families to understand the chance of passing the condition to future children.

Also known as:

Key symptoms:

Drooping of one or both upper eyelids (ptosis)Fusion of lower back and sacral vertebraeReduced flexibility or stiffness in the lower backPossible vision problems due to eyelid droopingShort stature in some casesPossible mild spinal abnormalitiesPossible difficulty with certain physical movements

Clinical phenotype terms (6)— hover any for plain English
Posterior fusion of lumbosacral vertebraeHP:0005626
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

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 Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome.

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No specialists are currently listed for Posterior fusion of lumbosacral vertebrae-blepharoptosis 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 Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome.

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.How severe is the eyelid drooping, and does my child need surgery to correct it?,What does the spinal imaging show, and are there any risks to the spinal cord or nerves?,Should we pursue genetic testing to look for the underlying cause?,What physical activities are safe, and are there any that should be avoided?,How often should we schedule follow-up visits for the eyes and spine?,Is there a risk of this condition being passed on to future children?,Are there any signs or symptoms I should watch for that would need urgent medical attention?

Common questions about Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome

What is Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome?

Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome is an extremely rare genetic condition that combines abnormalities of the lower spine with drooping of the upper eyelids (called blepharoptosis or ptosis). In this syndrome, some of the bones in the lower back and sacral area (the lumbosacral vertebrae) are fused together at the back, meaning they are joined when they should normally be separate. At the same time, affected individuals have drooping eyelids, which can affect one or both eyes and may interfere with vision if severe. This condition falls under a group of disorders

How is Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome inherited?

Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome typically begin?

Typical onset of Posterior fusion of lumbosacral vertebrae-blepharoptosis syndrome is neonatal. Age of onset can vary across affected individuals.