OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature

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ORPHA:371064
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8Treatment centers

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Overview

This entry refers to a group of congenital disorders of glycosylation (CDG) that are not linked to the X chromosome and where intellectual disability is a major feature. Congenital disorders of glycosylation are a large family of rare inherited metabolic conditions. In these disorders, the body has trouble attaching sugar molecules (glycans) to proteins and fats, a process called glycosylation. This process is essential for many body functions, including brain development, immune function, and organ growth. It is important to note that this particular Orphanet classification (371064) is now marked as OBSOLETE, meaning it has been retired as a grouping category. Patients previously classified under this umbrella term have likely been reclassified into more specific CDG subtypes as genetic knowledge has advanced. The intellectual disability seen in these conditions can range from mild to severe and is often accompanied by other neurological problems such as seizures, low muscle tone, and developmental delays. Other body systems, including the liver, heart, eyes, and digestive system, may also be affected depending on the specific underlying genetic cause. Treatment for CDG subtypes is mostly supportive, focusing on managing symptoms such as seizures, feeding difficulties, and developmental delays. A small number of CDG subtypes have specific treatments, such as mannose supplementation for PMM2-CDG in some cases, but for most types, no curative therapy exists. Early diagnosis and multidisciplinary care are important to optimize quality of life.

Also known as:

Key symptoms:

Intellectual disabilityDevelopmental delaysLow muscle tone (floppiness)SeizuresDifficulty feeding or failure to thriveAbnormal fat distribution or unusual fat padsLiver problemsEye abnormalities such as crossed eyesHeart defectsAbnormal blood clottingSkeletal abnormalitiesSpeech and language delaysPoor coordination or balance problemsDigestive issues such as diarrhea or vomiting

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

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 OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature.

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No actively recruiting trials found for OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature at this time.

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Search ClinicalTrials.gov ↗Join the OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature community →

No specialists are currently listed for OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability 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 OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature.

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Community

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Latest news about OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

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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 type of CDG does my child have, and what gene is affected?,Are there any targeted treatments available for this specific CDG subtype?,What organs should be monitored regularly, and how often should we schedule check-ups?,What therapies (physical, occupational, speech) would benefit my child the most right now?,Are there any clinical trials or research studies we should consider?,What emergency signs should I watch for at home, and when should I go to the emergency room?,Can you connect us with a CDG specialist center or patient support group?

Common questions about OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature

What is OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature?

This entry refers to a group of congenital disorders of glycosylation (CDG) that are not linked to the X chromosome and where intellectual disability is a major feature. Congenital disorders of glycosylation are a large family of rare inherited metabolic conditions. In these disorders, the body has trouble attaching sugar molecules (glycans) to proteins and fats, a process called glycosylation. This process is essential for many body functions, including brain development, immune function, and organ growth. It is important to note that this particular Orphanet classification (371064) is now m

How is OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature inherited?

OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature typically begin?

Typical onset of OBSOLETE: Non-X-linked congenital disorder of glycosylation with intellectual disability as a major feature is infantile. Age of onset can vary across affected individuals.