Microcephaly-brachydactyly-kyphoscoliosis syndrome

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:3433Q87.8
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

Microcephaly-brachydactyly-kyphoscoliosis syndrome is an extremely rare genetic condition that affects multiple body systems. The name describes its three main features: microcephaly (a smaller-than-expected head size, which often reflects reduced brain growth), brachydactyly (unusually short fingers and toes), and kyphoscoliosis (an abnormal curvature of the spine that combines both a forward rounding and a sideways curve). People with this syndrome typically also experience intellectual disability, short stature, and distinctive facial features. The condition is present from birth, and the combination of skeletal and neurological problems can significantly affect a child's development and daily functioning. Because this syndrome is so rare, with only a handful of cases described in the medical literature, there is limited information about its full range of symptoms and long-term outcomes. There is currently no cure or disease-specific treatment. Management focuses on addressing individual symptoms through supportive care, which may include physical therapy, orthopedic interventions for spinal problems, special education services, and regular monitoring of growth and development. A team of specialists working together is usually needed to provide the best care for affected individuals.

Also known as:

Key symptoms:

Smaller than normal head sizeShort fingers and toesAbnormal curvature of the spineIntellectual disabilityShort statureLow muscle toneDelayed developmental milestonesDistinctive facial featuresLow birth weightSeizures in some casesLimited joint movementSpeech and language delays

Clinical phenotype terms (19)— hover any for plain English
Shuffling gaitHP:0002362Decreased muscle massHP:0003199Flat occiputHP:0005469Atlantoaxial abnormalityHP:0003413Hypermobility of interphalangeal jointsHP:0005620Broad halluxHP:0010055Wide nasal ridgeHP:0012811
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 ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Microcephaly-brachydactyly-kyphoscoliosis syndrome.

View clinical trials →

No actively recruiting trials found for Microcephaly-brachydactyly-kyphoscoliosis syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Microcephaly-brachydactyly-kyphoscoliosis syndrome community →

No specialists are currently listed for Microcephaly-brachydactyly-kyphoscoliosis 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 Microcephaly-brachydactyly-kyphoscoliosis syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Microcephaly-brachydactyly-kyphoscoliosis syndromeForum →

No community posts yet. Be the first to share your experience with Microcephaly-brachydactyly-kyphoscoliosis syndrome.

Start the conversation →

Latest news about Microcephaly-brachydactyly-kyphoscoliosis syndrome

No recent news articles for Microcephaly-brachydactyly-kyphoscoliosis syndrome.

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.How severe is my child's condition compared to other reported cases?,What therapies should we start right away to support development?,How often should we monitor the spinal curvature, and when might surgery be needed?,Is genetic testing available that could confirm the diagnosis or help with family planning?,Are there any clinical trials or research studies we could participate in?,What educational supports should we request for our child at school?,What is the expected long-term outlook for my child's independence and quality of life?

Common questions about Microcephaly-brachydactyly-kyphoscoliosis syndrome

What is Microcephaly-brachydactyly-kyphoscoliosis syndrome?

Microcephaly-brachydactyly-kyphoscoliosis syndrome is an extremely rare genetic condition that affects multiple body systems. The name describes its three main features: microcephaly (a smaller-than-expected head size, which often reflects reduced brain growth), brachydactyly (unusually short fingers and toes), and kyphoscoliosis (an abnormal curvature of the spine that combines both a forward rounding and a sideways curve). People with this syndrome typically also experience intellectual disability, short stature, and distinctive facial features. The condition is present from birth, and the c

How is Microcephaly-brachydactyly-kyphoscoliosis syndrome inherited?

Microcephaly-brachydactyly-kyphoscoliosis syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Microcephaly-brachydactyly-kyphoscoliosis syndrome typically begin?

Typical onset of Microcephaly-brachydactyly-kyphoscoliosis syndrome is neonatal. Age of onset can vary across affected individuals.