Partial deletion of the long arm of chromosome 13 syndrome

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Overview

Partial deletion of the long arm of chromosome 13 syndrome (also known as 13q deletion syndrome or partial monosomy 13q) is a rare chromosomal disorder in which a portion of the long arm (q arm) of chromosome 13 is missing. The clinical presentation varies widely depending on the size and specific location of the deleted segment. This condition can affect multiple body systems, including the central nervous system, eyes, musculoskeletal system, and internal organs. Common features may include intellectual disability, developmental delay, growth retardation, microcephaly, and characteristic facial features such as a prominent nasal bridge, a short nose, and micrognathia. Eye abnormalities, including retinoblastoma (particularly when the deletion involves the 13q14 region encompassing the RB1 gene), coloboma, and microphthalmia, are well-recognized associations. Congenital heart defects, genitourinary anomalies, and limb malformations (notably thumb hypoplasia or aplasia) may also occur. The severity and spectrum of features depend heavily on which genes are lost within the deleted region. Deletions involving more proximal segments of 13q tend to produce different phenotypic profiles compared to more distal deletions. Some individuals with small deletions may have relatively mild manifestations, while larger deletions typically result in more significant multi-system involvement. Brain malformations, including holoprosencephaly, have been reported in some cases with specific deletion breakpoints. There is no cure for this condition, and management is supportive and symptom-based. Treatment may include early intervention programs, special education, physical and occupational therapy, surgical correction of congenital anomalies (such as heart defects or limb malformations), and ophthalmologic surveillance — particularly screening for retinoblastoma when the RB1 locus is involved. A multidisciplinary team approach involving geneticists, pediatricians, cardiologists, ophthalmologists, and developmental specialists is essential for optimal care.

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Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Partial deletion of the long arm of chromosome 13 syndrome.

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No specialists are currently listed for Partial deletion of the long arm of chromosome 13 syndrome.

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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

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Common questions about Partial deletion of the long arm of chromosome 13 syndrome

What is Partial deletion of the long arm of chromosome 13 syndrome?

Partial deletion of the long arm of chromosome 13 syndrome (also known as 13q deletion syndrome or partial monosomy 13q) is a rare chromosomal disorder in which a portion of the long arm (q arm) of chromosome 13 is missing. The clinical presentation varies widely depending on the size and specific location of the deleted segment. This condition can affect multiple body systems, including the central nervous system, eyes, musculoskeletal system, and internal organs. Common features may include intellectual disability, developmental delay, growth retardation, microcephaly, and characteristic fac

How is Partial deletion of the long arm of chromosome 13 syndrome inherited?

Partial deletion of the long arm of chromosome 13 syndrome follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Partial deletion of the long arm of chromosome 13 syndrome typically begin?

Typical onset of Partial deletion of the long arm of chromosome 13 syndrome is neonatal. Age of onset can vary across affected individuals.