Congenital disorder of glycosylation with intestinal involvement

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Overview

Congenital disorder of glycosylation with intestinal involvement (CDG with intestinal involvement) is a rare inherited metabolic condition that affects how the body adds sugar chains (called glycans) to proteins. This process, known as glycosylation, is essential for many proteins to work properly throughout the body. When glycosylation does not work correctly, it can cause problems in many organs, but in this particular group of conditions, the intestines and digestive system are especially affected. Patients with this condition often experience chronic diarrhea, poor absorption of nutrients from food (malabsorption), failure to gain weight and grow properly, and protein-losing enteropathy (a condition where protein leaks out through the intestinal wall). These digestive problems can lead to malnutrition, dehydration, and developmental delays if not managed carefully. Other body systems may also be involved, including the liver, immune system, and nervous system, depending on the specific genetic cause. Treatment is mainly supportive and focuses on managing symptoms. This may include specialized nutrition support such as parenteral nutrition (nutrition given through a vein), dietary modifications, and close monitoring of growth and development. In some rare subtypes, specific sugar supplements may help improve glycosylation. Because this is a group of related conditions rather than a single disease, the exact symptoms, severity, and outlook can vary widely depending on the underlying genetic defect.

Also known as:

Key symptoms:

Chronic diarrheaPoor weight gain and failure to thriveMalabsorption of nutrients from foodProtein-losing enteropathy (loss of protein through the gut)VomitingAbdominal bloating or distensionSwelling of the body due to low protein levels (edema)Developmental delayLiver problemsFrequent infections due to immune system issuesLow blood sugar episodesBlood clotting abnormalitiesDehydrationFeeding difficulties in infancyGrowth delay

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 ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Congenital disorder of glycosylation with intestinal involvement.

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No actively recruiting trials found for Congenital disorder of glycosylation with intestinal involvement at this time.

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No specialists are currently listed for Congenital disorder of glycosylation with intestinal involvement.

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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 Congenital disorder of glycosylation with intestinal involvement.

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Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific type of CDG does my child have, and which gene is affected?,Is there a targeted treatment available for this specific subtype?,What nutritional support does my child need, and will parenteral nutrition be necessary?,What are the warning signs that should prompt an emergency room visit?,How will this condition affect my child's growth and development over time?,Are there clinical trials or research studies we should consider?,What other organs should be monitored, and how often do we need follow-up testing?

Common questions about Congenital disorder of glycosylation with intestinal involvement

What is Congenital disorder of glycosylation with intestinal involvement?

Congenital disorder of glycosylation with intestinal involvement (CDG with intestinal involvement) is a rare inherited metabolic condition that affects how the body adds sugar chains (called glycans) to proteins. This process, known as glycosylation, is essential for many proteins to work properly throughout the body. When glycosylation does not work correctly, it can cause problems in many organs, but in this particular group of conditions, the intestines and digestive system are especially affected. Patients with this condition often experience chronic diarrhea, poor absorption of nutrients

How is Congenital disorder of glycosylation with intestinal involvement inherited?

Congenital disorder of glycosylation with intestinal involvement follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Congenital disorder of glycosylation with intestinal involvement typically begin?

Typical onset of Congenital disorder of glycosylation with intestinal involvement is neonatal. Age of onset can vary across affected individuals.