Alazami syndrome

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ORPHA:319671OMIM:615071Q87.1
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Overview

Alazami syndrome is a very rare genetic condition that primarily affects growth and brain development. It is sometimes also called LARP7-related primordial dwarfism or Alazami-Yuan syndrome. Children with this condition are noticeably shorter than their peers from early life, a pattern called primordial dwarfism, meaning the growth restriction begins before birth and continues afterward. In addition to short stature, most individuals with Alazami syndrome have intellectual disability that ranges from mild to severe, along with a distinctive facial appearance that may include a triangular face, prominent forehead, and deep-set eyes. The syndrome is caused by changes (mutations) in the LARP7 gene, which plays an important role in how cells process genetic information. Because this gene is involved in fundamental cell processes, its disruption affects multiple body systems. Some affected individuals may also have skeletal differences, delayed speech, and behavioral challenges. There is currently no cure for Alazami syndrome. Treatment focuses on managing symptoms and supporting development. This typically includes early intervention programs, speech therapy, occupational therapy, physical therapy, and special education services. Growth hormone therapy has been tried in some cases, but its effectiveness for this specific condition is not well established. Regular monitoring by a team of specialists helps ensure that children receive the support they need to reach their full potential.

Also known as:

Key symptoms:

Very short stature present from birth (primordial dwarfism)Intellectual disability (mild to severe)Delayed speech and language developmentSmall head size (microcephaly)Triangular-shaped faceProminent foreheadDeep-set eyesLow birth weightDelayed motor milestones like sitting and walkingThin body buildBehavioral difficultiesLearning difficultiesSkeletal abnormalitiesFeeding difficulties in infancy

Clinical phenotype terms (31)— hover any for plain English
Short palpebral fissureHP:0012745Mild microcephalyHP:0040196Narrow palpebral fissureHP:0045025Abnormal eating behaviorHP:0100738
Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

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 Alazami syndrome.

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No actively recruiting trials found for Alazami syndrome at this time.

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No specialists are currently listed for Alazami 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 Alazami syndrome.

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Community

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Latest news about Alazami syndrome

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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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 is the severity of my child's intellectual disability, and what therapies are recommended?,Should we consider growth hormone therapy, and what are the realistic expectations?,What early intervention services should we start, and how often?,Are there any other health complications we should screen for regularly?,What educational accommodations should we request at school?,Should other family members be tested to see if they are carriers?,Are there any clinical trials or research studies we could participate in?

Common questions about Alazami syndrome

What is Alazami syndrome?

Alazami syndrome is a very rare genetic condition that primarily affects growth and brain development. It is sometimes also called LARP7-related primordial dwarfism or Alazami-Yuan syndrome. Children with this condition are noticeably shorter than their peers from early life, a pattern called primordial dwarfism, meaning the growth restriction begins before birth and continues afterward. In addition to short stature, most individuals with Alazami syndrome have intellectual disability that ranges from mild to severe, along with a distinctive facial appearance that may include a triangular face,

How is Alazami syndrome inherited?

Alazami syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Alazami syndrome typically begin?

Typical onset of Alazami syndrome is neonatal. Age of onset can vary across affected individuals.