Macrocephaly-developmental delay syndrome

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ORPHA:397612OMIM:615637Q75.3
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Overview

Macrocephaly-developmental delay syndrome is a rare genetic condition characterized by an unusually large head size (macrocephaly) combined with delays in reaching developmental milestones such as sitting, walking, and talking. The condition is present from birth or early infancy and belongs to a group of disorders that affect brain growth and development. Children with this syndrome typically have a head circumference that is significantly larger than average for their age and sex. Along with the large head, affected individuals often experience intellectual disability of varying degrees, delayed speech and language development, and delays in motor skills like crawling and walking. Some children may also have distinctive facial features, low muscle tone (hypotonia), and behavioral challenges. Seizures can occur in some cases. The treatment landscape for macrocephaly-developmental delay syndrome is primarily supportive and symptom-based. There is currently no cure for the condition. Early intervention programs including speech therapy, occupational therapy, and physical therapy can help children reach their fullest potential. Regular monitoring by a team of specialists is important to track development and address any complications as they arise. Some children may benefit from special education services and behavioral support programs.

Key symptoms:

Unusually large head sizeDelayed speech and language developmentDelayed motor milestones like walking and sittingIntellectual disabilityLow muscle toneLearning difficultiesDistinctive facial featuresSeizures in some casesBehavioral challengesPoor coordinationFeeding difficulties in infancyTall stature or overgrowth in some cases

Clinical phenotype terms (26)— hover any for plain English
Abnormal speech discriminationHP:0001963
Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Macrocephaly-developmental delay syndrome.

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No actively recruiting trials found for Macrocephaly-developmental delay syndrome at this time.

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No specialists are currently listed for Macrocephaly-developmental delay 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 Macrocephaly-developmental delay syndrome.

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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.What is the specific genetic cause of my child's condition, and what does it mean for their future?,What therapies and early intervention services do you recommend starting right away?,Should we be concerned about seizures, and what signs should we watch for?,How often should my child's head size and brain be monitored with imaging?,Are there any clinical trials or new treatments being studied for this condition?,What is the chance that future children could also be affected?,What educational supports and accommodations should we request for school?

Common questions about Macrocephaly-developmental delay syndrome

What is Macrocephaly-developmental delay syndrome?

Macrocephaly-developmental delay syndrome is a rare genetic condition characterized by an unusually large head size (macrocephaly) combined with delays in reaching developmental milestones such as sitting, walking, and talking. The condition is present from birth or early infancy and belongs to a group of disorders that affect brain growth and development. Children with this syndrome typically have a head circumference that is significantly larger than average for their age and sex. Along with the large head, affected individuals often experience intellectual disability of varying degrees, de

At what age does Macrocephaly-developmental delay syndrome typically begin?

Typical onset of Macrocephaly-developmental delay syndrome is infantile. Age of onset can vary across affected individuals.