OBSOLETE: Total spina bifida cystica

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ORPHA:268748
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13Active trials8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Total spina bifida cystica is a severe form of spina bifida, which is a birth defect where the bones of the spine (vertebrae) do not form properly around the spinal cord. In this condition, the defect affects the entire length of the spine, and a fluid-filled sac (cyst) containing spinal cord tissue and nerves pushes through the opening in the back. This is one of the most severe types of neural tube defects, which are problems that happen very early in pregnancy when the brain and spinal cord are first forming. Because the defect involves the whole spine, this condition causes very serious problems including paralysis of the legs, loss of bladder and bowel control, and often hydrocephalus (a buildup of fluid in the brain). Babies born with this condition typically have significant neurological damage. The exposed or protruding spinal tissue is also at high risk for infection. Note that this Orphanet entry is marked as 'OBSOLETE,' meaning the classification has been updated or merged into other, more current diagnostic categories. Patients and families should work with their medical team to identify the most current terminology and classification for their specific condition. Treatment generally involves surgery shortly after birth to close the spinal defect, management of hydrocephalus with a shunt, and ongoing supportive care from a multidisciplinary team. Despite advances in treatment, the extensive nature of this defect means outcomes are often very serious.

Key symptoms:

Visible sac or opening along the entire back at birthComplete paralysis of the legsLoss of bladder controlLoss of bowel controlBuildup of fluid in the brain (hydrocephalus)Abnormal curvature of the spineLack of sensation below the defectDifficulty breathingRisk of brain infection (meningitis)Clubfoot or other foot deformitiesIntellectual disability in some casesSkin breakdown and pressure soresPoor growth and feeding difficulties

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 ↗

FDA & Trial Timeline

10 events
Nov 2025Transcutaneous Tibial Nerve Stimulation (TTNS) Treatment in Spina Bifida Pediatric Patients With Neurogenic Bladder

University of Texas Southwestern Medical Center — NA

TrialRECRUITING
Aug 2025Transcutaneous Spinal Stimulation for Children and Youth With Spina Bifida

Mayo Clinic — NA

TrialRECRUITING
Aug 2025Autologous Human Umbilical Cord Tissue Patch for Postnatal Closure of Open Neural Tube Defects

The University of Texas Health Science Center, Houston — NA

TrialRECRUITING
Mar 2025Prenatal and Postnatal Ultrasonographic Evaluation of Myelomeningocele to Predict Post-Surgical Outcomes

Superior University

TrialACTIVE NOT RECRUITING
Dec 2024QUALAS Validation in Dutch

Erasmus Medical Center

TrialRECRUITING
Sep 2024In Utero Surgery for Fetal Myelomeningocele: Decision-making Mechanisms and Psychological Impact of Prenatal Therapy

Assistance Publique - Hôpitaux de Paris — NA

TrialRECRUITING
Jun 2024Psychosocial Teleassistance Programme for Adults With Spina Bifida

University of Deusto — NA

TrialRECRUITING
Jan 2024Evaluation of muLtimodal and Non-invasive SPINa Bifida Neurovessels During Prospective Follow-up

Rennes University Hospital — NA

TrialRECRUITING
Nov 2023Operating Room Extubation and Postoperative Outcomes in Neonates With Meningomyelocele

Gaziantep City Hospital

TrialACTIVE NOT RECRUITING
Feb 2023Impact of Standing Programs in Children With Spina Bifida: A Single Subject Design

University of St. Augustine for Health Sciences — NA

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for OBSOLETE: Total spina bifida cystica.

13 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

13 recruitingView all trials with filters →
N/A9 trials
Transcutaneous Spinal Stimulation for Children and Youth With Spina Bifida
N/A
Actively Recruiting
PI: Kristin Zhao, Ph.D. (Mayo Clinic) · Sites: Rochester, Minnesota · Age: 518 yrs
Incontinence and Quality of Life in Children With Spina Bifida
N/A
Active
· Sites: Indianapolis, Indiana · Age: 899 yrs
Impact of Standing Programs in Children With Spina Bifida: A Single Subject Design
N/A
Actively Recruiting
· Sites: San Diego, California · Age: 512 yrs
Autologous Human Umbilical Cord Tissue Patch for Postnatal Closure of Open Neural Tube Defects
N/A
Actively Recruiting
PI: Stephen Fletcher, DO (The University of Texas Health Science Center, Hou) · Sites: Houston, Texas
Transcutaneous Tibial Nerve Stimulation (TTNS) Treatment in Spina Bifida Pediatric Patients With Neurogenic Bladder
N/A
Actively Recruiting
PI: Richard Adams, MD (University of Texas Southwestern Medical Center) · Sites: Dallas, Texas · Age: 818 yrs
Evaluation of muLtimodal and Non-invasive SPINa Bifida Neurovessels During Prospective Follow-up
N/A
Actively Recruiting
· Sites: Rennes, CHU de Rennes · Age: 699 yrs
Grand Valley State University (GVSU) Skills on Wheels
N/A
Active
· Sites: Grand Rapids, Michigan · Age: 517 yrs
In Utero Surgery for Fetal Myelomeningocele: Decision-making Mechanisms and Psychological Impact of Prenatal Therapy
N/A
Actively Recruiting
· Sites: Paris, Paris · Age: 1899 yrs
Psychosocial Teleassistance Programme for Adults With Spina Bifida
N/A
Actively Recruiting
· Sites: Bilbao, Bizkaia · Age: 1875 yrs
Other4 trials
Operating Room Extubation and Postoperative Outcomes in Neonates With Meningomyelocele
Active
PI: Recep Karakaşoğlu, MD (Gaziantep City Hospital) · Sites: Gaziantep, Gaziantep
Psychosocial Adjustment of Adolescents With Spina Bifida
Active
PI: Grayson N Holmbeck, PhD (Loyola University Chicago) · Sites: Chicago, Illinois · Age: 815 yrs
Prenatal and Postnatal Ultrasonographic Evaluation of Myelomeningocele to Predict Post-Surgical Outcomes
Active
· Sites: Faisalābad, Punjab Province
QUALAS Validation in Dutch
Actively Recruiting
PI: Lisette 't Hoen, MD, PhD (Erasmus Medical Center) · Sites: Groningen; Rotterdam · Age: 899 yrs

No specialists are currently listed for OBSOLETE: Total spina bifida cystica.

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 OBSOLETE: Total spina bifida cystica.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What is the full extent of my child's spinal defect and how does it affect their nervous system?,Will my child need a shunt for hydrocephalus, and what are the signs of shunt problems?,What surgeries will be needed and when should they happen?,What level of mobility and independence can we expect with proper treatment?,How should we manage bladder and bowel care at home?,Are there clinical trials or new treatments we should know about?,What is the risk of this happening again in a future pregnancy, and how can we reduce that risk?

Common questions about OBSOLETE: Total spina bifida cystica

What is OBSOLETE: Total spina bifida cystica?

Total spina bifida cystica is a severe form of spina bifida, which is a birth defect where the bones of the spine (vertebrae) do not form properly around the spinal cord. In this condition, the defect affects the entire length of the spine, and a fluid-filled sac (cyst) containing spinal cord tissue and nerves pushes through the opening in the back. This is one of the most severe types of neural tube defects, which are problems that happen very early in pregnancy when the brain and spinal cord are first forming. Because the defect involves the whole spine, this condition causes very serious p

How is OBSOLETE: Total spina bifida cystica inherited?

OBSOLETE: Total spina bifida cystica follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does OBSOLETE: Total spina bifida cystica typically begin?

Typical onset of OBSOLETE: Total spina bifida cystica is neonatal. Age of onset can vary across affected individuals.

Are there clinical trials for OBSOLETE: Total spina bifida cystica?

Yes — 13 recruiting clinical trials are currently listed for OBSOLETE: Total spina bifida cystica on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.