Congenital disorder of glycosylation with skin involvement

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Overview

Congenital disorder of glycosylation with skin involvement (CDG with skin involvement) is a rare inherited metabolic condition that affects how the body adds sugar chains (called glycans) to proteins and fats. This process, known as glycosylation, is essential for many body functions. When glycosylation does not work properly, it can affect multiple organs, but in this particular group of disorders, the skin is notably affected. Patients with this condition may experience a range of skin problems such as unusually loose or wrinkled skin (cutis laxa), dry or scaly skin (ichthyosis), skin that is easily damaged or slow to heal, and abnormal fat distribution under the skin. Beyond the skin, other body systems can also be involved, including the brain, liver, heart, and immune system. Symptoms can vary widely depending on the specific genetic cause. There is currently no cure for congenital disorders of glycosylation with skin involvement. Treatment focuses on managing symptoms and supporting affected organ systems. Some patients may benefit from specific dietary supplements such as mannose or galactose depending on the underlying defect, though these therapies are only effective in certain subtypes. A team of specialists is usually needed to provide comprehensive care. Early diagnosis through genetic testing is important to guide treatment and monitor for complications.

Also known as:

Key symptoms:

Loose or wrinkled skin (cutis laxa)Dry, scaly skin (ichthyosis)Skin that bruises or tears easilyAbnormal fat distribution under the skinDelayed growth and developmentIntellectual disability or learning difficultiesLow muscle tone (floppiness)Liver problemsSeizuresFeeding difficulties in infancySkeletal abnormalitiesImmune system problems with frequent infectionsAbnormal blood clottingVision problemsHeart defects

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

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

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

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

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 Congenital disorder of glycosylation with skin involvement.

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Community

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Caregiver Resources

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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 specific subtype of CDG does my child have, and which gene is affected?,Are there any dietary supplements or specific treatments that could help this subtype?,What organs should be monitored regularly, and how often should we schedule check-ups?,Are there any activities or medications we should avoid because of this condition?,What should we do in case of a medical emergency related to this condition?,Are there clinical trials or research studies we could participate in?,What is the expected course of this condition, and how might it change over time?

Common questions about Congenital disorder of glycosylation with skin involvement

What is Congenital disorder of glycosylation with skin involvement?

Congenital disorder of glycosylation with skin involvement (CDG with skin involvement) is a rare inherited metabolic condition that affects how the body adds sugar chains (called glycans) to proteins and fats. This process, known as glycosylation, is essential for many body functions. When glycosylation does not work properly, it can affect multiple organs, but in this particular group of disorders, the skin is notably affected. Patients with this condition may experience a range of skin problems such as unusually loose or wrinkled skin (cutis laxa), dry or scaly skin (ichthyosis), skin that