Interstitial lung disease due to SP-C deficiency

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ORPHA:440392OMIM:610913J84.8
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Overview

Interstitial lung disease due to SP-C deficiency (also called surfactant protein C deficiency or SFTPC-related interstitial lung disease) is a rare inherited lung condition caused by changes in the SFTPC gene. This gene provides instructions for making surfactant protein C, a substance that helps keep the tiny air sacs in the lungs (called alveoli) open and working properly. When this protein is missing or abnormal, the lungs become inflamed and scarred over time, making it harder to breathe. The disease can affect people of any age, from newborns to adults, though it often first appears in infancy or early childhood. The main problem is that the lungs gradually lose their ability to transfer oxygen into the blood. This leads to symptoms like persistent coughing, rapid or labored breathing, and low oxygen levels. Over time, the lung tissue becomes stiff and scarred, a process called fibrosis. There is currently no cure for this condition. Treatment focuses on managing symptoms, reducing lung inflammation, and supporting breathing. Doctors may use corticosteroids, hydroxychloroquine, or azithromycin to try to slow the disease. In severe cases, supplemental oxygen or even a lung transplant may be needed. Because this is such a rare disease, treatment is largely based on small studies and expert opinion rather than large clinical trials.

Also known as:

Key symptoms:

Persistent dry or wet coughFast or labored breathing (especially in infants)Low oxygen levels in the bloodShortness of breath with activity or even at restBluish tint to the lips or fingertips (cyanosis)Poor weight gain or failure to thrive in infantsFatigue and low energyCrackling sounds in the lungs heard by a doctorClubbing of the fingers (widening and rounding of fingertips) over timeRecurrent respiratory infections

Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

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 Interstitial lung disease due to SP-C deficiency.

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No actively recruiting trials found for Interstitial lung disease due to SP-C deficiency at this time.

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

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No specialists are currently listed for Interstitial lung disease due to SP-C deficiency.

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 Interstitial lung disease due to SP-C deficiency.

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Community

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Latest news about Interstitial lung disease due to SP-C deficiency

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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.What stage is my (or my child's) lung disease, and how quickly might it progress?,Which medications do you recommend, and what are the potential side effects?,Should other family members be tested for the SFTPC gene change?,When should we consider a lung transplant evaluation, and what does that process involve?,Are there any clinical trials or research studies we could participate in?,How often should we have follow-up visits and lung function tests?,What signs should prompt us to go to the emergency room immediately?

Common questions about Interstitial lung disease due to SP-C deficiency

What is Interstitial lung disease due to SP-C deficiency?

Interstitial lung disease due to SP-C deficiency (also called surfactant protein C deficiency or SFTPC-related interstitial lung disease) is a rare inherited lung condition caused by changes in the SFTPC gene. This gene provides instructions for making surfactant protein C, a substance that helps keep the tiny air sacs in the lungs (called alveoli) open and working properly. When this protein is missing or abnormal, the lungs become inflamed and scarred over time, making it harder to breathe. The disease can affect people of any age, from newborns to adults, though it often first appears in i

How is Interstitial lung disease due to SP-C deficiency inherited?

Interstitial lung disease due to SP-C deficiency follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.