Saccular spinal dysraphism with a stalk to the dome

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ORPHA:645319
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Overview

Saccular spinal dysraphism with a stalk to the dome is an extremely rare type of spinal birth defect that falls under the broader category of neural tube defects (also called spinal dysraphisms). In this condition, during early development in the womb, the spinal column does not close properly, leading to a sac-like outpouching (called a saccular lesion) from the spine. What makes this specific subtype distinct is that the sac has a narrow stalk connecting it to its rounded top (the dome). This stalk-to-dome connection is an important feature that doctors use to classify and plan treatment for this type of defect. Spinal dysraphisms can affect the spinal cord, the surrounding membranes (meninges), and nearby nerves. Depending on the location and severity, this condition may cause problems with movement, sensation, bladder and bowel control, and in some cases can lead to infection or other complications if the sac is open or leaking spinal fluid. The condition is typically identified at birth or even before birth through prenatal ultrasound or MRI imaging. Treatment usually involves surgical repair to close the defect and protect the spinal cord and nerves. The goal of surgery is to prevent infection, preserve as much nerve function as possible, and manage any associated complications such as hydrocephalus (excess fluid in the brain). Long-term follow-up with multiple specialists is often needed to address ongoing neurological, urological, and orthopedic needs. Outcomes vary widely depending on the size, location, and contents of the sac.

Key symptoms:

Visible sac or lump on the back at birthWeakness or paralysis in the legsLoss of sensation in the lower bodyDifficulty controlling the bladderDifficulty controlling the bowelsLeaking of spinal fluid from the backHydrocephalus (fluid buildup in the brain)Foot or leg deformities such as clubfootSkin changes or dimple over the spineRecurrent urinary tract infectionsTethered spinal cord symptoms

Inheritance

Multifactorial

Caused by a mix of several genes and environmental factors

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Saccular spinal dysraphism with a stalk to the dome.

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No actively recruiting trials found for Saccular spinal dysraphism with a stalk to the dome at this time.

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No specialists are currently listed for Saccular spinal dysraphism with a stalk to the dome.

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 Saccular spinal dysraphism with a stalk to the dome.

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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.

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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 exactly is inside the sac, and how does that affect my child's outlook?,Will my child need surgery, and when should it be done?,Is there a risk of hydrocephalus, and how will it be monitored?,What kind of bladder and bowel management will my child need?,What therapies (physical, occupational) should we start, and when?,Are there signs of tethered cord that I should watch for as my child grows?,Should our family have genetic testing or counseling for future pregnancies?

Common questions about Saccular spinal dysraphism with a stalk to the dome

What is Saccular spinal dysraphism with a stalk to the dome?

Saccular spinal dysraphism with a stalk to the dome is an extremely rare type of spinal birth defect that falls under the broader category of neural tube defects (also called spinal dysraphisms). In this condition, during early development in the womb, the spinal column does not close properly, leading to a sac-like outpouching (called a saccular lesion) from the spine. What makes this specific subtype distinct is that the sac has a narrow stalk connecting it to its rounded top (the dome). This stalk-to-dome connection is an important feature that doctors use to classify and plan treatment for

How is Saccular spinal dysraphism with a stalk to the dome inherited?

Saccular spinal dysraphism with a stalk to the dome follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Saccular spinal dysraphism with a stalk to the dome typically begin?

Typical onset of Saccular spinal dysraphism with a stalk to the dome is neonatal. Age of onset can vary across affected individuals.