Brain malformation-congenital heart disease-postaxial polydactyly syndrome

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Overview

Brain malformation-congenital heart disease-postaxial polydactyly syndrome is a rare condition present from birth that affects several parts of the body at the same time. It is sometimes called BMCHP syndrome. The three main features are structural problems in the brain, heart defects that are present at birth, and extra fingers or toes on the outer (pinky) side of the hands or feet — a feature called postaxial polydactyly. Because the brain, heart, and limbs are all affected, this is considered a multi-system condition. Children born with this syndrome may have a wide range of symptoms depending on how severely each organ system is affected. Brain malformations can lead to intellectual disability, developmental delays, seizures, and problems with muscle tone. Heart defects vary but can be serious and may require surgery shortly after birth. The extra digits on the hands or feet are usually the most visible sign and are often noticed immediately at delivery. There is currently no cure for this syndrome. Treatment focuses on managing each symptom separately. Heart defects may be repaired through surgery. Seizures can often be controlled with medication. Children typically need ongoing support from a team of specialists, as well as physical, occupational, and speech therapy to help them reach their full potential.

Also known as:

Key symptoms:

Extra fingers or toes on the outer (pinky) side of the hand or footStructural brain abnormalities present from birthHeart defects present from birthIntellectual disability or learning difficultiesDevelopmental delays (sitting, walking, talking later than expected)Seizures or epilepsyLow or unusually high muscle toneSmall head size (microcephaly) in some casesFeeding difficulties in infancyVision or eye problems in some casesBreathing difficulties related to heart or brain problems

Clinical phenotype terms (24)— hover any for plain English
Brittle hairHP:0002299Pulmonary artery stenosisHP:0004415Aplasia/Hypoplasia of the cerebellar vermisHP:0006817
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

1 available

XARELTO

RIVAROXABAN· Janssen Pharmaceuticals, Inc.■ Boxed Warning

for thromboprophylaxis in pediatric patients 2 years and older with congenital heart disease after the Fontan procedure

No actively recruiting trials found for Brain malformation-congenital heart disease-postaxial polydactyly syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Brain malformation-congenital heart disease-postaxial polydactyly syndrome community →

No specialists are currently listed for Brain malformation-congenital heart disease-postaxial polydactyly 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 Brain malformation-congenital heart disease-postaxial polydactyly syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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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 specific brain malformation does my child have, and what does that mean for their development?,Does my child's heart defect need surgery, and if so, when?,Should we have genetic testing done, and what type of test is most likely to find the cause?,What early intervention therapies should we start right away?,What signs should prompt me to go to the emergency room?,Are there any clinical trials or research studies we could participate in?,What support services or patient organizations are available for families dealing with this condition?

Common questions about Brain malformation-congenital heart disease-postaxial polydactyly syndrome

What is Brain malformation-congenital heart disease-postaxial polydactyly syndrome?

Brain malformation-congenital heart disease-postaxial polydactyly syndrome is a rare condition present from birth that affects several parts of the body at the same time. It is sometimes called BMCHP syndrome. The three main features are structural problems in the brain, heart defects that are present at birth, and extra fingers or toes on the outer (pinky) side of the hands or feet — a feature called postaxial polydactyly. Because the brain, heart, and limbs are all affected, this is considered a multi-system condition. Children born with this syndrome may have a wide range of symptoms depen

How is Brain malformation-congenital heart disease-postaxial polydactyly syndrome inherited?

Brain malformation-congenital heart disease-postaxial polydactyly syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Brain malformation-congenital heart disease-postaxial polydactyly syndrome typically begin?

Typical onset of Brain malformation-congenital heart disease-postaxial polydactyly syndrome is neonatal. Age of onset can vary across affected individuals.