Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome

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ORPHA:444077OMIM:616368Q87.8
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8Treatment centers

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

Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome, sometimes referred to by its abbreviation CHOPS syndrome, is a rare genetic condition that affects many parts of the body at the same time. The name describes the main features doctors look for: learning and thinking difficulties, distinctive facial features that may appear broader or rougher than typical, heart problems present from birth, a tendency toward obesity, breathing or lung issues, shorter-than-average height, and abnormalities in the bones and skeleton. This condition is caused by a change (mutation) in a specific gene that plays an important role in how cells read and use genetic instructions during development. Because this gene is active in many tissues throughout the body, problems can show up in multiple organ systems. Babies may be identified shortly after birth due to heart defects or distinctive facial features, though the full picture of the syndrome often becomes clearer during early childhood as developmental delays become more apparent. There is currently no cure for CHOPS syndrome. Care focuses on managing each symptom individually — for example, heart surgery for structural heart defects, respiratory support for lung problems, educational support for learning difficulties, and physical or occupational therapy to help with movement and daily skills. A team of specialists working together gives patients the best chance at a good quality of life.

Also known as:

Key symptoms:

Intellectual disability or learning difficultiesDistinctive facial features including a broad or coarse appearanceHeart defects present from birth (congenital heart disease)Obesity or significant weight gainBreathing problems or lung involvementShort statureBone and skeletal abnormalitiesDelayed speech and language developmentFeeding difficulties in infancyHearing lossVision problemsRecurrent respiratory infections

Clinical phenotype terms (50)— hover any for plain English
Abnormal skeletal morphologyHP:0011842
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 ↗

FDA & Trial Timeline

3 events
May 2024CHOP Plus Mosunetuzumab as First Line in Patients With Richter´s Syndrome: a Phase II Study of the Spanish Group of CLL

GELLC (Grupo Español de Leucemia Linfocítica Crónica) — PHASE2

TrialRECRUITING
Jul 2019A Trial of CHOP-R Therapy, With or Without Acalabrutinib, in Patients With Newly Diagnosed Richter's Syndrome

University of Birmingham — PHASE2

TrialRECRUITING
Mar 2017A Phase II Study of Venetoclax in Combination With Dose-adjusted EPOCH-R or R-CHOP for Patients With Richter's Syndrome

Dana-Farber Cancer Institute — PHASE2

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome.

View clinical trials →

No actively recruiting trials found for Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome community →

No specialists are currently listed for Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia 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 Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome

Disease timeline:

New recruiting trial: A Trial of CHOP-R Therapy, With or Without Acalabrutinib, in Patients With Newly Diagnosed Richter's Syndrome

A new clinical trial is recruiting patients for Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome

New recruiting trial: CHOP Plus Mosunetuzumab as First Line in Patients With Richter´s Syndrome: a Phase II Study of the Spanish Group of CLL

A new clinical trial is recruiting patients for Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome

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.Which specific mutation was found in the AFF4 gene, and what does that mean for my child's outlook?,What heart tests does my child need, and how often should we follow up with a cardiologist?,What early intervention therapies should we start right away, and how do we access them?,Are there any clinical trials or research studies we could participate in?,What signs of breathing problems should prompt us to go to the emergency room?,How can we best support my child's learning and communication development?,Should other family members be tested, and what is the chance of this happening again in a future pregnancy?

Common questions about Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome

What is Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome?

Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome, sometimes referred to by its abbreviation CHOPS syndrome, is a rare genetic condition that affects many parts of the body at the same time. The name describes the main features doctors look for: learning and thinking difficulties, distinctive facial features that may appear broader or rougher than typical, heart problems present from birth, a tendency toward obesity, breathing or lung issues, shorter-than-average height, and abnormalities in the bones and skeleton. This co

How is Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome inherited?

Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome typically begin?

Typical onset of Cognitive impairment-coarse facies-heart defects-obesity-pulmonary involvement-short stature-skeletal dysplasia syndrome is neonatal. Age of onset can vary across affected individuals.