SHOX-related short stature

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ORPHA:314795OMIM:300582Q87.1
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Overview

SHOX-related short stature is a genetic condition caused by changes in or near the SHOX gene (Short Stature Homeobox-containing gene), which plays an important role in bone growth, especially in the long bones of the arms and legs. This condition is one of the most common genetic causes of short stature. People with SHOX-related short stature are shorter than expected for their family, and the degree of short stature can range from mild to more significant. Some individuals may also develop a condition called Madelung deformity, where the wrist bones grow unevenly, causing the wrist to look bent or curved. Other features can include shorter forearms and lower legs compared to the upper arms and thighs, a wider and more stocky body build, and increased carrying angle of the elbows. The condition affects both males and females, though females tend to be more noticeably affected because they lack a second functional copy of the SHOX gene on their X chromosome. Treatment with growth hormone therapy has been approved and can help increase final adult height, especially when started early in childhood. SHOX-related short stature includes a spectrum that ranges from isolated short stature to Léri-Weill dyschondrosteosis, which involves more pronounced skeletal features. The condition is also sometimes referred to as SHOX deficiency or SHOX haploinsufficiency.

Key symptoms:

Short stature (shorter than expected for family height)Disproportionately short forearmsDisproportionately short lower legsMadelung deformity (curved or bent wrists)Increased carrying angle of the elbows (arms angle outward)Stocky or muscular body buildShort hands and feetWrist pain or limited wrist movementBowing of the forearm bonesHigh-arched palateShort neckDelayed growth during childhood and puberty

Clinical phenotype terms (16)— hover any for plain English
Tibial bowingHP:0002982Forearm undergrowthHP:0009821Madelung deformityHP:0003067Lower limb undergrowthHP:0009816Ulnar radial head dislocationHP:0005856Episodic ketoacidosisHP:0005974
Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for SHOX-related short stature.

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No actively recruiting trials found for SHOX-related short stature at this time.

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No specialists are currently listed for SHOX-related short stature.

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 SHOX-related short stature.

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Community

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Latest news about SHOX-related short stature

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.How severe is my child's SHOX deficiency, and what final adult height can we expect with and without treatment?,Is growth hormone therapy recommended, and when should we start?,How will we monitor for Madelung deformity, and what signs should I watch for?,Are there any side effects of growth hormone therapy I should know about?,Should other family members be tested for SHOX deficiency?,Will my child need any orthopedic evaluations or treatments?,How often should we have follow-up appointments to track growth progress?

Common questions about SHOX-related short stature

What is SHOX-related short stature?

SHOX-related short stature is a genetic condition caused by changes in or near the SHOX gene (Short Stature Homeobox-containing gene), which plays an important role in bone growth, especially in the long bones of the arms and legs. This condition is one of the most common genetic causes of short stature. People with SHOX-related short stature are shorter than expected for their family, and the degree of short stature can range from mild to more significant. Some individuals may also develop a condition called Madelung deformity, where the wrist bones grow unevenly, causing the wrist to look be

At what age does SHOX-related short stature typically begin?

Typical onset of SHOX-related short stature is childhood. Age of onset can vary across affected individuals.