Rhizomelic dysplasia, Patterson-Lowry type

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ORPHA:2831OMIM:601438Q78.8
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Overview

Rhizomelic dysplasia, Patterson-Lowry type (also called Patterson-Lowry rhizomelic dysplasia) is an extremely rare genetic bone disorder that primarily affects the growth and development of the skeleton. The term "rhizomelic" refers to shortening of the bones closest to the body, meaning the upper arms (humeri) and upper legs (femora) are disproportionately short compared to the rest of the limbs. This condition was first described by Patterson and Lowry and is distinct from other forms of rhizomelic skeletal dysplasia. People with this condition typically show short stature with particularly shortened upper limbs and thighs, along with other skeletal abnormalities that may include changes in the shape of the vertebrae and other bones. Facial features may also be affected. The condition is usually apparent at birth or in early childhood because of the noticeable limb shortening and growth delays. Because this condition is so rare, with only a handful of cases described in the medical literature, the treatment landscape is limited. There is currently no cure or disease-specific therapy. Management focuses on supportive care, including orthopedic interventions to address skeletal complications, physical therapy to maximize mobility and function, and regular monitoring of growth and development. A team of specialists is typically needed to provide comprehensive care.

Key symptoms:

Short statureShortened upper arms and upper legs (rhizomelic limb shortening)Disproportionate body proportionsAbnormal bone development visible on X-raysChanges in the shape of the spine bonesBroad or unusual facial featuresLimited range of motion in jointsDelayed motor milestonesBowing of the long bonesShort hands and feet

Clinical phenotype terms (17)— hover any for plain English
Deviation of fingerHP:0004097Deformed humeral headsHP:0005687Short humerusHP:0005792Large faceHP:0100729
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 Rhizomelic dysplasia, Patterson-Lowry type.

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No actively recruiting trials found for Rhizomelic dysplasia, Patterson-Lowry type at this time.

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No specialists are currently listed for Rhizomelic dysplasia, Patterson-Lowry type.

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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 Rhizomelic dysplasia, Patterson-Lowry type.

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Community

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Caregiver Resources

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Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific skeletal abnormalities does my child have, and how might they change over time?,Should we pursue genetic testing, and what type of testing do you recommend?,What physical therapy or rehabilitation programs would be most helpful?,Are there any orthopedic surgeries that might improve my child's mobility or comfort?,How often should we schedule follow-up imaging and specialist visits?,Are there any complications we should watch for as my child grows?,Can you connect us with other families or support groups for rare skeletal conditions?

Common questions about Rhizomelic dysplasia, Patterson-Lowry type

What is Rhizomelic dysplasia, Patterson-Lowry type?

Rhizomelic dysplasia, Patterson-Lowry type (also called Patterson-Lowry rhizomelic dysplasia) is an extremely rare genetic bone disorder that primarily affects the growth and development of the skeleton. The term "rhizomelic" refers to shortening of the bones closest to the body, meaning the upper arms (humeri) and upper legs (femora) are disproportionately short compared to the rest of the limbs. This condition was first described by Patterson and Lowry and is distinct from other forms of rhizomelic skeletal dysplasia. People with this condition typically show short stature with particularly

How is Rhizomelic dysplasia, Patterson-Lowry type inherited?

Rhizomelic dysplasia, Patterson-Lowry type follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Rhizomelic dysplasia, Patterson-Lowry type typically begin?

Typical onset of Rhizomelic dysplasia, Patterson-Lowry type is neonatal. Age of onset can vary across affected individuals.