Immunodeficiency due to MASP-2 deficiency

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ORPHA:331187OMIM:613791D84.1
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Overview

Immunodeficiency due to MASP-2 deficiency is a rare inherited condition that affects part of the body's immune system called the complement system. The complement system is a group of proteins in the blood that help fight infections by recognizing and destroying bacteria, viruses, and other harmful germs. MASP-2 (Mannan-binding lectin Serine Protease 2) is a key protein in one branch of this system called the lectin pathway. When MASP-2 is missing or not working properly, the lectin pathway cannot activate correctly, leaving the body more vulnerable to certain infections. People with MASP-2 deficiency may experience repeated or severe bacterial infections, particularly pneumonia, skin infections, and bloodstream infections (sepsis). Some individuals may also develop autoimmune-like symptoms, where the immune system mistakenly attacks the body's own tissues. However, the severity of symptoms can vary widely — some people with MASP-2 deficiency have frequent serious infections, while others may have few or no noticeable problems throughout their lives. Treatment focuses on preventing and managing infections. This may include antibiotics given promptly at the first sign of infection, and in some cases, long-term preventive (prophylactic) antibiotics. Vaccinations are also important to help protect against common bacterial infections. There is currently no cure or specific therapy that replaces the missing MASP-2 protein, so management is mainly supportive and aimed at reducing the risk and impact of infections.

Key symptoms:

Frequent or severe bacterial infectionsRepeated pneumonia or lung infectionsSkin infections or abscessesBloodstream infections (sepsis)Infections caused by bacteria that have a protective capsule (encapsulated bacteria)Inflammatory or autoimmune-like symptomsChronic fatigue related to recurrent illnessSlow recovery from infectionsPossible inflammatory skin rashes

Inheritance

Autosomal recessive

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

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Immunodeficiency due to MASP-2 deficiency.

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No actively recruiting trials found for Immunodeficiency due to MASP-2 deficiency at this time.

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No specialists are currently listed for Immunodeficiency due to MASP-2 deficiency.

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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 Immunodeficiency due to MASP-2 deficiency.

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Community

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Caregiver Resources

NORD Caregiver Resources

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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.How severe is my (or my child's) MASP-2 deficiency, and what level of infection risk does it carry?,Should I (or my child) take preventive antibiotics on a daily basis?,Are there specific vaccines that are especially important for this condition?,What are the warning signs that an infection is becoming serious and needs emergency care?,Should other family members be tested for MASP-2 deficiency?,Are there any activities or environments I should avoid to reduce infection risk?,Are there any clinical trials or new treatments being studied for complement deficiencies?

Common questions about Immunodeficiency due to MASP-2 deficiency

What is Immunodeficiency due to MASP-2 deficiency?

Immunodeficiency due to MASP-2 deficiency is a rare inherited condition that affects part of the body's immune system called the complement system. The complement system is a group of proteins in the blood that help fight infections by recognizing and destroying bacteria, viruses, and other harmful germs. MASP-2 (Mannan-binding lectin Serine Protease 2) is a key protein in one branch of this system called the lectin pathway. When MASP-2 is missing or not working properly, the lectin pathway cannot activate correctly, leaving the body more vulnerable to certain infections. People with MASP-2 d

How is Immunodeficiency due to MASP-2 deficiency inherited?

Immunodeficiency due to MASP-2 deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.