Nijmegen breakage syndrome-like disorder

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ORPHA:240760OMIM:613078Q87.8
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Overview

Nijmegen breakage syndrome-like disorder (NBS-like disorder) is an extremely rare genetic condition that shares many features with Nijmegen breakage syndrome (NBS) but is caused by a different gene. This condition affects the body's ability to repair damaged DNA, which is essential for keeping cells healthy. Because of this DNA repair problem, affected individuals typically show a small head size (microcephaly), growth delays, a weakened immune system, and an increased risk of developing cancer, particularly lymphomas and leukemias. Children with NBS-like disorder may have distinctive facial features, including a receding forehead and prominent midface. They often experience recurrent infections due to their compromised immune system. Intellectual development can be affected to varying degrees. The condition also causes increased sensitivity to radiation, which is important to know before any medical imaging or cancer treatment involving radiation. There is currently no cure for NBS-like disorder. Treatment focuses on managing symptoms, preventing and treating infections, monitoring for cancer, and providing supportive care. Immunoglobulin replacement therapy may be used for immune deficiency. Cancer surveillance is a critical part of ongoing care. Because of the DNA repair defect, standard cancer treatments may need to be modified to avoid excessive toxicity from radiation or certain chemotherapy drugs.

Also known as:

Key symptoms:

Unusually small head size (microcephaly)Short stature and growth delaysRecurrent infections due to weak immune systemDistinctive facial features such as receding forehead and prominent midfaceIncreased risk of cancer, especially lymphoma and leukemiaIntellectual disability or learning difficultiesSensitivity to radiationLow levels of antibodies (immunoglobulin deficiency)Chromosomal instability seen in lab testsBird-like facial appearanceDelayed developmentSkin changes such as café-au-lait spots or vitiligo

Inheritance

Autosomal recessive

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

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

Treatments

2 available

Abilify

aripiprazole· Otsuka Pharmaceutical Development■ Boxed Warning

Acute Treatment of Manic and Mixed Episodes associated with Bipolar I Disorder

Provigil

Modafinil· Cephalon, Inc.

indicated to improve wakefulness in adult patients with excessive sleepiness associated with shift work disorder (SWD)

No actively recruiting trials found for Nijmegen breakage syndrome-like disorder at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Nijmegen breakage syndrome-like disorder community →

No specialists are currently listed for Nijmegen breakage syndrome-like disorder.

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

Financial Resources

1 resources

Midazolam

Roche

Anxiety Disorder

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Travel Grants

No travel grants are currently matched to Nijmegen breakage syndrome-like disorder.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Nijmegen breakage syndrome-like disorder

No recent news articles for Nijmegen breakage syndrome-like disorder.

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Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

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.How often should my child be screened for cancer, and what tests are involved?,Is immunoglobulin replacement therapy recommended, and how will it be given?,Are there any vaccines my child should avoid because of their immune deficiency?,What precautions should we take regarding radiation exposure during medical procedures?,What developmental support services would benefit my child?,If cancer develops, how will treatment be modified to account for the DNA repair defect?,Are there any clinical trials or research studies we should know about?

Common questions about Nijmegen breakage syndrome-like disorder

What is Nijmegen breakage syndrome-like disorder?

Nijmegen breakage syndrome-like disorder (NBS-like disorder) is an extremely rare genetic condition that shares many features with Nijmegen breakage syndrome (NBS) but is caused by a different gene. This condition affects the body's ability to repair damaged DNA, which is essential for keeping cells healthy. Because of this DNA repair problem, affected individuals typically show a small head size (microcephaly), growth delays, a weakened immune system, and an increased risk of developing cancer, particularly lymphomas and leukemias. Children with NBS-like disorder may have distinctive facial

How is Nijmegen breakage syndrome-like disorder inherited?

Nijmegen breakage syndrome-like disorder follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Nijmegen breakage syndrome-like disorder typically begin?

Typical onset of Nijmegen breakage syndrome-like disorder is childhood. Age of onset can vary across affected individuals.

What treatment and support options exist for Nijmegen breakage syndrome-like disorder?

1 patient support program are currently tracked on UniteRare for Nijmegen breakage syndrome-like disorder. See the treatments and support programs sections for copay assistance, eligibility, and contact details.