Spondyloepimetaphyseal dysplasia, Bieganski type

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:168448
Who is this for?
Show terms as
8Treatment centers

Where are you in your journey?

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

Overview

Spondyloepimetaphyseal dysplasia, Bieganski type (also called SEMD Bieganski type) is an extremely rare inherited bone disorder that affects the growth and development of the skeleton. The name describes which parts of the bones are affected: the spine (spondylo-), the ends of long bones (epi-), and the areas near the growth plates (metaphyseal). This condition was first described by Dr. Bieganski and colleagues, and only a handful of cases have been reported in the medical literature. People with this condition typically have short stature that becomes noticeable in early childhood. The bones of the spine, arms, and legs do not develop normally, leading to skeletal abnormalities that can be seen on X-rays. Common features include short limbs, a short trunk, bowed legs, and abnormalities of the vertebrae (bones of the spine). Some affected individuals may also have distinctive facial features and intellectual disability. Because this condition is so rare, there is no specific cure or targeted treatment available. Management focuses on addressing symptoms as they arise, which may include orthopedic interventions for bone and joint problems, physical therapy to maintain mobility, and supportive care. Regular monitoring by a team of specialists is important to manage complications and optimize quality of life.

Key symptoms:

Short statureShort arms and legsShort trunkBowed legsAbnormal curvature of the spineFlattened vertebrae (bones of the spine)Irregular growth plates visible on X-rayWaddling gait or difficulty walkingJoint stiffness or limited range of motionDistinctive facial featuresIntellectual disability in some casesDelayed motor development

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Spondyloepimetaphyseal dysplasia, Bieganski type.

View clinical trials →

No actively recruiting trials found for Spondyloepimetaphyseal dysplasia, Bieganski type at this time.

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

Search ClinicalTrials.gov ↗Join the Spondyloepimetaphyseal dysplasia, Bieganski type community →

No specialists are currently listed for Spondyloepimetaphyseal dysplasia, Bieganski type.

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 Spondyloepimetaphyseal dysplasia, Bieganski type.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Spondyloepimetaphyseal dysplasia, Bieganski typeForum →

No community posts yet. Be the first to share your experience with Spondyloepimetaphyseal dysplasia, Bieganski type.

Start the conversation →

Latest news about Spondyloepimetaphyseal dysplasia, Bieganski type

No recent news articles for Spondyloepimetaphyseal dysplasia, Bieganski type.

Follow this condition to be notified when news becomes available.

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.What specific skeletal abnormalities does my child have, and how might they change over time?,Is genetic testing available to confirm the diagnosis, and should other family members be tested?,What orthopedic treatments or surgeries might be needed in the future?,How often should we schedule follow-up X-rays and specialist visits?,Are there physical therapy programs that specialize in skeletal dysplasias?,What signs of complications should I watch for at home?,Are there any research studies or registries we can participate in?

Common questions about Spondyloepimetaphyseal dysplasia, Bieganski type

What is Spondyloepimetaphyseal dysplasia, Bieganski type?

Spondyloepimetaphyseal dysplasia, Bieganski type (also called SEMD Bieganski type) is an extremely rare inherited bone disorder that affects the growth and development of the skeleton. The name describes which parts of the bones are affected: the spine (spondylo-), the ends of long bones (epi-), and the areas near the growth plates (metaphyseal). This condition was first described by Dr. Bieganski and colleagues, and only a handful of cases have been reported in the medical literature. People with this condition typically have short stature that becomes noticeable in early childhood. The bone

How is Spondyloepimetaphyseal dysplasia, Bieganski type inherited?

Spondyloepimetaphyseal dysplasia, Bieganski type follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Spondyloepimetaphyseal dysplasia, Bieganski type typically begin?

Typical onset of Spondyloepimetaphyseal dysplasia, Bieganski type is childhood. Age of onset can vary across affected individuals.