Classic glucose transporter type 1 deficiency syndrome

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ORPHA:71277OMIM:606777E74.8
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1Active trials5Specialists8Treatment centers

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

Classic glucose transporter type 1 deficiency syndrome, also known as Glut1 DS, De Vivo disease, or GLUT1 deficiency, is a rare inherited metabolic disorder. It is caused by a problem with a protein called GLUT1, which normally carries glucose (sugar) from the blood into the brain. Without enough GLUT1 working properly, the brain does not get enough fuel to function well. This leads to a range of neurological problems that usually begin in infancy or early childhood. The most common symptoms include seizures that start in the first year of life, delays in reaching developmental milestones like sitting and walking, difficulty with movement and coordination, and intellectual disability ranging from mild to severe. Some children also have a movement disorder that causes involuntary muscle stiffness or writhing movements, especially when they are hungry or tired. The good news is that a special high-fat, low-carbohydrate diet called the ketogenic diet is an effective treatment for many people with this condition. This diet gives the brain an alternative fuel source called ketones, which do not need GLUT1 to enter the brain. When started early, the ketogenic diet can significantly reduce seizures and improve development. While there is no cure, many people with GLUT1 deficiency can lead meaningful lives with proper management and support.

Also known as:

Key symptoms:

Seizures starting in infancy, often resistant to standard seizure medicationsDevelopmental delays, including delayed speech and languageIntellectual disability, ranging from mild to moderateDifficulty with coordination and balance (ataxia)Involuntary muscle movements or stiffness (movement disorder)Abnormal eye movements, such as rapid back-and-forth eye movements (opsoclonus)Symptoms that get worse when fasting or hungryFatigue and low energy, especially before mealsSpasticity (muscle stiffness)Behavioral problems or attention difficultiesHeadachesConfusion or altered alertness, especially when hungry

Clinical phenotype terms (33)— hover any for plain English
Abnormal erythrocyte morphologyHP:0001877HypoglycorrhachiaHP:0011972
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

2 events
May 2026A Phase II Study With Exploratory Outcomes of Fucose Supplementation in GLUT1 Deficiency Syndrome

Oregon Health and Science University — PHASE2

TrialNOT YET RECRUITING
Jan 2023Harmonic Ratio in Patients With GLUT1 Deficiency Syndrome

IRCCS National Neurological Institute "C. Mondino" Foundation

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Classic glucose transporter type 1 deficiency syndrome.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Other1 trial
Harmonic Ratio in Patients With GLUT1 Deficiency Syndrome
Active
PI: Roberto De Icco (IRCCS, Mondino Foundation) · Sites: Pavia; Pavia · Age: 360 yrs

Specialists

5 foundView all specialists →
GP
Gerard Vockley, MD, PhD
PITTSBURGH, PA
Specialist
PI on 5 active trials
JP
Juan M. Pascual, MD, PhD
PACE, FL
Specialist
PI on 2 active trials
SS
Sylvia Stockler
Specialist
PI on 1 active trial
RS
Rodrigo T. Starosta
Specialist
PI on 1 active trial
RI
Roberto De Icco
Specialist
PI on 1 active trial

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 Classic glucose transporter type 1 deficiency syndrome.

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Community

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Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

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Family & Caregiver Grants

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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.Is the ketogenic diet the right treatment for my child right now, and how soon should we start?,Which anti-seizure medications should we avoid because of GLUT1 deficiency?,How will we know if the ketogenic diet is working, and how long should we try it before deciding if it is effective?,What developmental therapies do you recommend, and how often should my child receive them?,Are there any clinical trials or newer treatments we should consider?,What should I do if my child has a prolonged seizure — do we need an emergency medication at home?,As my child grows into adulthood, how will the management of this condition change?

Common questions about Classic glucose transporter type 1 deficiency syndrome

What is Classic glucose transporter type 1 deficiency syndrome?

Classic glucose transporter type 1 deficiency syndrome, also known as Glut1 DS, De Vivo disease, or GLUT1 deficiency, is a rare inherited metabolic disorder. It is caused by a problem with a protein called GLUT1, which normally carries glucose (sugar) from the blood into the brain. Without enough GLUT1 working properly, the brain does not get enough fuel to function well. This leads to a range of neurological problems that usually begin in infancy or early childhood. The most common symptoms include seizures that start in the first year of life, delays in reaching developmental milestones lik

How is Classic glucose transporter type 1 deficiency syndrome inherited?

Classic glucose transporter type 1 deficiency syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Classic glucose transporter type 1 deficiency syndrome typically begin?

Typical onset of Classic glucose transporter type 1 deficiency syndrome is infantile. Age of onset can vary across affected individuals.

Are there clinical trials for Classic glucose transporter type 1 deficiency syndrome?

Yes — 1 recruiting clinical trial is currently listed for Classic glucose transporter type 1 deficiency syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Classic glucose transporter type 1 deficiency syndrome?

5 specialists and care centers treating Classic glucose transporter type 1 deficiency syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.