B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome

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ORPHA:536467OMIM:615349Q79.6
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Overview

B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome (also called spEDS-B3GALT6, or sometimes referred to as EDSSPD2 or spondylodysplastic EDS type 2) is a rare inherited connective tissue disorder caused by changes (mutations) in the B3GALT6 gene. Connective tissue is like the body's scaffolding — it holds bones, skin, joints, and organs together. When this gene does not work properly, the body cannot build a key sugar chain (called a glycosaminoglycan) that is essential for strong, flexible connective tissue. This leads to problems throughout the body. People with this condition are often born with or develop short stature, weak muscle tone (hypotonia), very flexible or unstable joints, and skin that is stretchy, fragile, or heals poorly. The spine and other bones may be shaped differently than usual, which is why 'spondylodysplastic' (meaning abnormal bone development) is part of the name. Some individuals also experience delayed development, intellectual disability, and eye problems. There is currently no cure for this condition. Treatment focuses on managing symptoms, protecting joints, supporting development, and preventing complications. A team of specialists works together to help patients live as comfortably and independently as possible. Early diagnosis is important because it allows families to plan the right therapies and monitoring from the start.

Also known as:

Key symptoms:

Short stature or slow growthWeak muscle tone (floppy muscles), especially in infancyVery flexible, loose, or unstable jointsSkin that is stretchy, thin, or fragilePoor wound healing or unusual scarringAbnormal spine shape (such as scoliosis or kyphosis)Bone shape differences visible on X-rayDelayed motor development (sitting, walking later than expected)Intellectual disability or learning difficulties (in some patients)Eye problems such as myopia (nearsightedness) or corneal issuesFlat feet or other foot deformitiesJoint pain or frequent joint dislocationsSmall hands and feetDental problems including crowded or fragile teeth

Clinical phenotype terms (50)— hover any for plain English
Soft, doughy skinHP:0001027Spondyloepimetaphyseal dysplasiaHP:0002651Metaphyseal wideningHP:0003016Slender long bones with narrow diaphysesHP:0004993Large joint dislocationsHP:0005008Acetabular dysplasiaHP:0008807Dysplasia of the femoral headHP:0010575Multiple joint dislocationHP:0012095
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 B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome.

View clinical trials →

No actively recruiting trials found for B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome community →

No specialists are currently listed for B3GALT6-related spondylodysplastic Ehlers-Danlos 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 B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about B3GALT6-related spondylodysplastic Ehlers-Danlos 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.Which specialists should be part of my care team, and how often should I see each one?,What specific signs of spinal instability or cord compression should I watch for, and when should I go to the emergency room?,Should other family members be tested for the B3GALT6 gene mutation?,What physical activities are safe, and which ones should I avoid to protect my joints?,Are there any clinical trials or research studies I could participate in?,What educational or developmental supports should my child receive?,How will this condition change over time, and what complications should we plan for?

Common questions about B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome

What is B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome?

B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome (also called spEDS-B3GALT6, or sometimes referred to as EDSSPD2 or spondylodysplastic EDS type 2) is a rare inherited connective tissue disorder caused by changes (mutations) in the B3GALT6 gene. Connective tissue is like the body's scaffolding — it holds bones, skin, joints, and organs together. When this gene does not work properly, the body cannot build a key sugar chain (called a glycosaminoglycan) that is essential for strong, flexible connective tissue. This leads to problems throughout the body. People with this condition are of

How is B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome inherited?

B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome typically begin?

Typical onset of B3GALT6-related spondylodysplastic Ehlers-Danlos syndrome is neonatal. Age of onset can vary across affected individuals.