SLC39A8-CDG

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ORPHA:468699OMIM:616721E77.8
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10Specialists8Treatment centers

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

SLC39A8-CDG (also known as SLC39A8-related congenital disorder of glycosylation, or CDG type IIn) is an extremely rare inherited metabolic condition caused by changes (mutations) in the SLC39A8 gene. This gene provides instructions for making a protein that helps transport manganese and other metals into cells. Manganese is essential for many enzymes to work properly, including those involved in glycosylation — the process of attaching sugar molecules to proteins. When this transporter does not work correctly, manganese levels in the blood drop very low, and glycosylation becomes impaired, leading to problems throughout the body. Children with SLC39A8-CDG typically present in infancy with a range of serious symptoms. These can include intellectual disability, seizures, short stature, hearing loss, and vision problems. Many affected children have skeletal abnormalities such as dwarfism and cranial malformations. Low muscle tone (hypotonia) and movement disorders, including dystonia, are also common. Some children develop Leigh-like syndrome, a severe brain condition. The treatment landscape for SLC39A8-CDG has shown promise with manganese supplementation, which can partially restore manganese levels and improve glycosylation. Galactose supplementation has also been used alongside manganese in some patients. While these treatments have led to meaningful improvements in some individuals, the condition remains serious and requires ongoing management by a team of specialists.

Also known as:

Key symptoms:

Intellectual disabilitySeizuresShort stature or dwarfismLow muscle tone (floppiness)Movement problems such as dystoniaHearing lossVision problemsCranial (skull) abnormalitiesSkeletal abnormalitiesDelayed developmentDifficulty with balance and coordinationFeeding difficultiesRecurrent infectionsBrain abnormalities on MRI (Leigh-like pattern)Failure to thrive

Clinical phenotype terms (41)— hover any for plain English
Severe muscular hypotoniaHP:0006829Abnormal blood zinc concentrationHP:0008277Type II transferrin isoform profileHP:0012301Profound global developmental delayHP:0012736HypomanganesemiaHP:0032098Visual fixation instabilityHP:0025405
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 ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for SLC39A8-CDG.

View clinical trials →

No actively recruiting trials found for SLC39A8-CDG at this time.

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

Search ClinicalTrials.gov ↗Join the SLC39A8-CDG community →

Specialists

10 foundView all specialists →
EP
Eva Morava-Kozicz, MD, PhD
ROCHESTER, MN
Specialist
PI on 7 active trials
LS
Luisa Sturiale
Specialist
1 SLC39A8-CDG publication
AP
Anna Maria Pinto
Specialist
1 SLC39A8-CDG publication
AR
Alessandra Renieri
Specialist
1 SLC39A8-CDG publication
BG
Barbara Garavaglia
Specialist
1 SLC39A8-CDG publication
EB
Eleonora Bonaventura
Specialist
1 SLC39A8-CDG publication
GC
Giovanni Cioni
Specialist
1 SLC39A8-CDG publication
RP
Rosa Pasquariello
Specialist
1 SLC39A8-CDG publication
MA
Maria Grazia Alessandrì
Specialist
1 SLC39A8-CDG publication
CP
Celeste Panteghini
Specialist
1 SLC39A8-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 SLC39A8-CDG.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about SLC39A8-CDG

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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 is the recommended dose of manganese supplementation for my child, and how will you monitor for safe levels?,Should galactose supplementation also be considered?,How often should blood tests and imaging be repeated to track progress?,What therapies (physical, occupational, speech) should we start, and how often?,Are there any clinical trials or research studies my child could participate in?,What is the expected developmental trajectory for my child given their specific mutations?,What emergency signs should I watch for, and what should I do if they occur?

Common questions about SLC39A8-CDG

What is SLC39A8-CDG?

SLC39A8-CDG (also known as SLC39A8-related congenital disorder of glycosylation, or CDG type IIn) is an extremely rare inherited metabolic condition caused by changes (mutations) in the SLC39A8 gene. This gene provides instructions for making a protein that helps transport manganese and other metals into cells. Manganese is essential for many enzymes to work properly, including those involved in glycosylation — the process of attaching sugar molecules to proteins. When this transporter does not work correctly, manganese levels in the blood drop very low, and glycosylation becomes impaired, lea

How is SLC39A8-CDG inherited?

SLC39A8-CDG follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does SLC39A8-CDG typically begin?

Typical onset of SLC39A8-CDG is infantile. Age of onset can vary across affected individuals.

Which specialists treat SLC39A8-CDG?

10 specialists and care centers treating SLC39A8-CDG are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.