ALG13-CDG

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ORPHA:324422OMIM:300884E77.8
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16Specialists8Treatment centers

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

ALG13-CDG (also known as ALG13-congenital disorder of glycosylation) is an extremely rare genetic condition that affects how the body adds sugar chains to proteins, a process called glycosylation. The ALG13 gene provides instructions for making a protein that plays an important role in the early steps of building these sugar chains. When this gene does not work properly, many proteins throughout the body cannot function correctly, leading to problems in multiple organ systems. The condition primarily affects the brain and nervous system. Most affected individuals experience epileptic seizures that often begin in infancy and can be very difficult to control. Intellectual disability and developmental delays are common, ranging from mild to severe. Some children may also have low muscle tone (floppiness), movement difficulties, and distinctive facial features. Other possible features include liver problems, immune system issues, and feeding difficulties. There is currently no cure for ALG13-CDG. Treatment focuses on managing symptoms, particularly controlling seizures with anti-epileptic medications, and providing supportive therapies such as physical therapy, occupational therapy, and speech therapy. Because the condition is so rare, knowledge about the full range of symptoms and best treatments is still growing. Research is ongoing to better understand this disorder and develop more targeted therapies.

Also known as:

Key symptoms:

Seizures that are hard to control (epilepsy)Intellectual disabilityDevelopmental delaysLow muscle tone (floppiness)Difficulty feedingSpeech and language delaysMovement and coordination problemsDistinctive facial featuresLiver problemsImmune system weaknessDelayed motor milestones like sitting and walkingAbnormal eye movementsMicrocephaly (smaller than average head size)Behavioral difficulties

Clinical phenotype terms (19)— hover any for plain English
Global brain atrophyHP:0002283Abnormal lateral ventricle morphologyHP:0030047
Inheritance

X-linked dominant

Carried on the X chromosome; a single copy can cause the condition

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for ALG13-CDG.

View clinical trials →

No actively recruiting trials found for ALG13-CDG at this time.

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

Search ClinicalTrials.gov ↗Join the ALG13-CDG community →

Specialists

16 foundView all specialists →
TK
Tamas Kozicz
Specialist
2 ALG13-CDG publications
EM
Eva Morava
Specialist
2 ALG13-CDG publications
RS
Rameen Shah
Specialist
2 ALG13-CDG publications
SR
Silvia Radenkovic
Specialist
2 ALG13-CDG publications
IS
Ibrahim Shammas
GLENDORA, CA
Specialist
2 ALG13-CDG publications
EP
Eva Morava-Kozicz, MD, PhD
ROCHESTER, MN
Specialist
PI on 7 active trials
AE
Andrew C Edmondson
TYLER, TX
Specialist
1 ALG13-CDG publication
RA
Ruqaiah Altassan
Specialist
1 ALG13-CDG publication
AB
Anna Bogdańska
Specialist
1 ALG13-CDG publication
EE
Erik A Eklund
RALEIGH, NC
Specialist
1 ALG13-CDG publication
MS
Mustafa Sadek
NAPLES, FL
Specialist
1 ALG13-CDG publication
SV
Sanne Verberkmoes
Specialist
1 ALG13-CDG publication
BN
Bobby G Ng
FOUNTAIN VALLEY, CA
Specialist
1 ALG13-CDG publication
HF
Hudson H Freeze
Specialist
1 ALG13-CDG publication
KM
Karolina Mitusińska
Specialist
1 ALG13-CDG publication
AG
Artur Góra
Specialist
1 ALG13-CDG publication

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 ALG13-CDG.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about ALG13-CDG

No recent news articles for ALG13-CDG.

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 type of seizures does my child have, and what is the best medication plan?,Should we consider the ketogenic diet for seizure control?,What developmental therapies should we start, and how often?,How often should we monitor liver function and other organ systems?,Are there any clinical trials or research studies we could participate in?,What is the expected developmental trajectory for my child based on their specific genetic variant?,What emergency plan should we have in place for prolonged seizures?

Common questions about ALG13-CDG

What is ALG13-CDG?

ALG13-CDG (also known as ALG13-congenital disorder of glycosylation) is an extremely rare genetic condition that affects how the body adds sugar chains to proteins, a process called glycosylation. The ALG13 gene provides instructions for making a protein that plays an important role in the early steps of building these sugar chains. When this gene does not work properly, many proteins throughout the body cannot function correctly, leading to problems in multiple organ systems. The condition primarily affects the brain and nervous system. Most affected individuals experience epileptic seizures

How is ALG13-CDG inherited?

ALG13-CDG follows a x-linked dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does ALG13-CDG typically begin?

Typical onset of ALG13-CDG is infantile. Age of onset can vary across affected individuals.

Which specialists treat ALG13-CDG?

16 specialists and care centers treating ALG13-CDG are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.