Rubinstein-Taybi syndrome due to 16p13.3 microdeletion

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ORPHA:353281OMIM:610543Q87.2
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Overview

Rubinstein-Taybi syndrome due to 16p13.3 microdeletion (sometimes called RTS or RSTS) is a rare genetic condition caused by a tiny missing piece of chromosome 16 in a region called 16p13.3. This deleted region typically includes the CREBBP gene, which plays an important role in how the body develops before and after birth. The syndrome affects multiple parts of the body and is usually recognized at birth or in early infancy. Children with this condition typically have distinctive facial features, including downward-slanting eyes, a beaked nose, and a grimacing smile. One of the hallmark signs is broad thumbs and big toes, which may also be angled. Most individuals have some degree of intellectual disability, ranging from mild to moderate, along with delayed growth and short stature. Many children also experience feeding difficulties in infancy and may have heart defects, kidney problems, or eye issues. There is currently no cure for Rubinstein-Taybi syndrome. Treatment focuses on managing individual symptoms and supporting development through early intervention programs, speech therapy, occupational therapy, and physical therapy. Regular monitoring by a team of specialists is essential to address the wide range of potential medical issues. With appropriate support, many individuals with this condition can lead fulfilling lives, though they typically need some level of ongoing assistance.

Key symptoms:

Broad thumbs and big toesIntellectual disabilityShort statureDistinctive facial features including a beaked noseDownward-slanting eyesFeeding difficulties in infancyDelayed speech and language developmentDelayed motor milestones like sitting and walkingHeart defects present at birthEye problems such as crossed eyes or blocked tear ductsKidney abnormalitiesConstipationDental crowding or extra teethIncreased risk of certain tumorsUndescended testicles in boys

Clinical phenotype terms (50)— hover any for plain English
AgoraphobiaHP:0000756
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 Rubinstein-Taybi syndrome due to 16p13.3 microdeletion.

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No actively recruiting trials found for Rubinstein-Taybi syndrome due to 16p13.3 microdeletion at this time.

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No specialists are currently listed for Rubinstein-Taybi syndrome due to 16p13.3 microdeletion.

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 Rubinstein-Taybi syndrome due to 16p13.3 microdeletion.

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Community

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Caregiver Resources

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Questions for your doctor

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  • Q1.What specific medical screenings does my child need and how often?,Are there any heart or kidney problems that need to be addressed right away?,What early intervention therapies should we start, and how do we access them?,What precautions should we take regarding anesthesia and surgery?,How should we monitor for tumor risk as my child grows?,What educational supports and accommodations should we request at school?,Are there any clinical trials or new research studies we should know about?

Common questions about Rubinstein-Taybi syndrome due to 16p13.3 microdeletion

What is Rubinstein-Taybi syndrome due to 16p13.3 microdeletion?

Rubinstein-Taybi syndrome due to 16p13.3 microdeletion (sometimes called RTS or RSTS) is a rare genetic condition caused by a tiny missing piece of chromosome 16 in a region called 16p13.3. This deleted region typically includes the CREBBP gene, which plays an important role in how the body develops before and after birth. The syndrome affects multiple parts of the body and is usually recognized at birth or in early infancy. Children with this condition typically have distinctive facial features, including downward-slanting eyes, a beaked nose, and a grimacing smile. One of the hallmark signs

How is Rubinstein-Taybi syndrome due to 16p13.3 microdeletion inherited?

Rubinstein-Taybi syndrome due to 16p13.3 microdeletion follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Rubinstein-Taybi syndrome due to 16p13.3 microdeletion typically begin?

Typical onset of Rubinstein-Taybi syndrome due to 16p13.3 microdeletion is neonatal. Age of onset can vary across affected individuals.