Congenital disorder of glycosylation with epilepsy as a major feature

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:371071
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 disorders of glycosylation (CDG) with epilepsy as a major feature are a group of rare inherited metabolic conditions where the body cannot properly attach sugar molecules (glycans) to proteins and fats. This process, called glycosylation, is essential for many body functions, including brain development and nervous system signaling. When glycosylation goes wrong, it can affect many organs, but in this particular group, seizures (epilepsy) are one of the most prominent and troublesome symptoms. Children with these conditions typically develop seizures early in life, often in infancy or early childhood. The seizures can be difficult to control with standard anti-seizure medications. Other common features may include developmental delay, intellectual disability, low muscle tone, movement problems, feeding difficulties, and sometimes abnormalities in other organs such as the liver or heart. The severity varies widely depending on the specific genetic cause. Treatment is mainly supportive and focused on controlling seizures, supporting development through therapies, and managing other organ involvement. For a small number of specific CDG subtypes, targeted treatments such as dietary sugar supplementation (for example, mannose in PMM2-CDG or galactose in certain types) may help. However, for most forms, there is no cure, and management focuses on improving quality of life. A team of specialists including neurologists, metabolic geneticists, and therapists is usually needed to provide comprehensive care.

Also known as:

Key symptoms:

Seizures that are hard to controlDevelopmental delayIntellectual disabilityLow muscle tone (floppy baby)Difficulty feeding or swallowingPoor weight gain or failure to thriveAbnormal eye movementsMovement problems or unsteady walkingSpeech delayLiver problemsAbnormal fat distribution or unusual fat padsInverted nipplesCrossed eyes (strabismus)Small head size (microcephaly)Episodes of stroke-like symptoms

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Congenital disorder of glycosylation with epilepsy as a major feature.

View clinical trials →

No actively recruiting trials found for Congenital disorder of glycosylation with epilepsy as a major feature at this time.

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

Search ClinicalTrials.gov ↗Join the Congenital disorder of glycosylation with epilepsy as a major feature community →

No specialists are currently listed for Congenital disorder of glycosylation with epilepsy as a major feature.

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 epilepsy as a major feature.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Congenital disorder of glycosylation with epilepsy as a major featureForum →

No community posts yet. Be the first to share your experience with Congenital disorder of glycosylation with epilepsy as a major feature.

Start the conversation →

Latest news about Congenital disorder of glycosylation with epilepsy as a major feature

No recent news articles for Congenital disorder of glycosylation with epilepsy as a major feature.

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 dietary supplements that could help my child's specific subtype?,What is the best seizure management plan, and when should I use rescue medication?,What developmental therapies should we start, and how often?,Are there clinical trials or research studies my child might be eligible for?,What other organs should be monitored, and how often do we need follow-up testing?,What does this diagnosis mean for future pregnancies and other family members?

Common questions about Congenital disorder of glycosylation with epilepsy as a major feature

What is Congenital disorder of glycosylation with epilepsy as a major feature?

Congenital disorders of glycosylation (CDG) with epilepsy as a major feature are a group of rare inherited metabolic conditions where the body cannot properly attach sugar molecules (glycans) to proteins and fats. This process, called glycosylation, is essential for many body functions, including brain development and nervous system signaling. When glycosylation goes wrong, it can affect many organs, but in this particular group, seizures (epilepsy) are one of the most prominent and troublesome symptoms. Children with these conditions typically develop seizures early in life, often in infancy

At what age does Congenital disorder of glycosylation with epilepsy as a major feature typically begin?

Typical onset of Congenital disorder of glycosylation with epilepsy as a major feature is infantile. Age of onset can vary across affected individuals.