Schuurs-Hoeijmakers syndrome

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ORPHA:329224OMIM:615009Q87.0
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Overview

Schuurs-Hoeijmakers syndrome, also known as PACS1-related syndrome or PACS1 neurodevelopmental disorder, is a rare genetic condition caused by a change (mutation) in the PACS1 gene. This syndrome affects multiple parts of the body and is characterized by intellectual disability, distinctive facial features, and developmental delays. Children with this condition typically have delays in reaching milestones like sitting, walking, and talking. The facial features often include a flat nasal bridge, widely spaced eyes, thin upper lip, and low-set ears. Many individuals also experience seizures, feeding difficulties in infancy, and behavioral challenges. Beyond the brain and face, Schuurs-Hoeijmakers syndrome can affect the heart, eyes, and digestive system. Some children are born with heart defects, and others may have vision problems or issues with bowel movements (such as constipation). Behavioral features can include autism spectrum traits, anxiety, and difficulty with social interactions. There is currently no cure for Schuurs-Hoeijmakers syndrome. Treatment focuses on managing individual symptoms and supporting development. This includes speech therapy, physical therapy, occupational therapy, and special education services. Seizures can often be managed with anti-seizure medications. Heart defects may require surgical repair. Early intervention and a team of specialists working together can significantly improve quality of life for affected individuals and their families.

Also known as:

Key symptoms:

Intellectual disability (usually mild to moderate)Delayed speech and language developmentDelayed motor milestones like walkingDistinctive facial featuresSeizures or epilepsyFeeding difficulties in infancyLow muscle tone (floppy muscles)Heart defects present at birthVision problems or crossed eyesBehavioral challenges including autism-like traitsConstipation or digestive issuesWidely spaced eyesFlat nasal bridgeSmall head sizeHearing problems

Clinical phenotype terms (50)— hover any for plain English
Oral aversionHP:0012523Abnormal temper tantrumsHP:0025160
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

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 Schuurs-Hoeijmakers syndrome.

View clinical trials →

No actively recruiting trials found for Schuurs-Hoeijmakers syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Schuurs-Hoeijmakers syndrome community →

No specialists are currently listed for Schuurs-Hoeijmakers 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 Schuurs-Hoeijmakers syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Schuurs-Hoeijmakers 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.What specific PACS1 mutation does my child have, and what does it mean for their development?,What therapies should we start right away, and how often should they occur?,Does my child need a heart evaluation or any other organ screening?,What should I do if my child has a seizure, and when should I call emergency services?,What educational supports and accommodations should I request at school?,Are there any clinical trials or research studies we could participate in?,What is the chance of having another child with this condition?

Common questions about Schuurs-Hoeijmakers syndrome

What is Schuurs-Hoeijmakers syndrome?

Schuurs-Hoeijmakers syndrome, also known as PACS1-related syndrome or PACS1 neurodevelopmental disorder, is a rare genetic condition caused by a change (mutation) in the PACS1 gene. This syndrome affects multiple parts of the body and is characterized by intellectual disability, distinctive facial features, and developmental delays. Children with this condition typically have delays in reaching milestones like sitting, walking, and talking. The facial features often include a flat nasal bridge, widely spaced eyes, thin upper lip, and low-set ears. Many individuals also experience seizures, fee

How is Schuurs-Hoeijmakers syndrome inherited?

Schuurs-Hoeijmakers syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Schuurs-Hoeijmakers syndrome typically begin?

Typical onset of Schuurs-Hoeijmakers syndrome is neonatal. Age of onset can vary across affected individuals.