Congenital disorder of glycosylation with developmental anomaly

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:371235
Who is this for?
Show terms as
8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Congenital disorder of glycosylation with developmental anomaly (CDG with developmental anomaly) is a rare inherited metabolic condition that affects how the body adds sugar chains (called glycans) to proteins and fats. These sugar chains are essential for many body functions, including proper development of organs and tissues before and after birth. When this process does not work correctly, it can lead to a wide range of developmental problems that may be visible at birth or become apparent during early childhood. This group of conditions can affect many different organ systems. Children born with this type of CDG may have structural birth defects (such as heart defects, skeletal abnormalities, or brain malformations), intellectual disability, delayed development, poor muscle tone, feeding difficulties, and failure to thrive. The severity varies widely depending on the specific genetic cause and which step in the glycosylation pathway is disrupted. There is currently no cure for most congenital disorders of glycosylation. Treatment is mainly supportive and focuses on managing individual symptoms. Some specific subtypes may respond to dietary supplements such as mannose or galactose, but this depends on the exact genetic diagnosis. A team of specialists is usually needed to provide comprehensive care. Early diagnosis through genetic testing is important to guide treatment and family planning.

Also known as:

Key symptoms:

Developmental delay or intellectual disabilityBirth defects affecting the heart, brain, or skeletonPoor muscle tone (floppy baby)Feeding difficulties in infancyFailure to thrive or poor weight gainSeizures or epilepsyAbnormal fat distribution or unusual skin appearanceCrossed eyes or other vision problemsLiver problems or elevated liver enzymesAbnormal blood clotting or easy bruisingSkeletal abnormalities such as curved spine or joint problemsSpeech and language delaysMovement problems or difficulty with coordinationHormonal imbalances

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

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 developmental anomaly.

View clinical trials →

No actively recruiting trials found for Congenital disorder of glycosylation with developmental anomaly at this time.

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

Search ClinicalTrials.gov ↗Join the Congenital disorder of glycosylation with developmental anomaly community →

No specialists are currently listed for Congenital disorder of glycosylation with developmental anomaly.

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 developmental anomaly.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Congenital disorder of glycosylation with developmental anomalyForum →

No community posts yet. Be the first to share your experience with Congenital disorder of glycosylation with developmental anomaly.

Start the conversation →

Latest news about Congenital disorder of glycosylation with developmental anomaly

No recent news articles for Congenital disorder of glycosylation with developmental anomaly.

Follow this condition to be notified when news becomes available.

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 specific type of CDG does my child have, and what gene is affected?,Are there any targeted treatments or supplements that could help my child's specific subtype?,What developmental milestones should we watch for, and when should we be concerned?,What specialists should be part of our care team, and how often should we see them?,Are there any clinical trials or research studies my child might be eligible for?,What emergency situations should I watch for, and what should I do if they occur?,Should other family members be tested, and what does this mean for future pregnancies?

Common questions about Congenital disorder of glycosylation with developmental anomaly

What is Congenital disorder of glycosylation with developmental anomaly?

Congenital disorder of glycosylation with developmental anomaly (CDG with developmental anomaly) is a rare inherited metabolic condition that affects how the body adds sugar chains (called glycans) to proteins and fats. These sugar chains are essential for many body functions, including proper development of organs and tissues before and after birth. When this process does not work correctly, it can lead to a wide range of developmental problems that may be visible at birth or become apparent during early childhood. This group of conditions can affect many different organ systems. Children bo

At what age does Congenital disorder of glycosylation with developmental anomaly typically begin?

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