Disorder of carbohydrate absorption and transport

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ORPHA:309001
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1Active trials8Treatment centers

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Overview

Disorder of carbohydrate absorption and transport is a group of rare inherited conditions in which the body has trouble absorbing or moving sugars and carbohydrates from the intestines into the bloodstream, or from the blood into the cells that need them. Carbohydrates are a major source of energy, and when they cannot be properly absorbed or transported, it can lead to a wide range of digestive and nutritional problems. This group includes several specific conditions such as glucose-galactose malabsorption, congenital sucrase-isomaltase deficiency, congenital lactase deficiency, and disorders of glucose transporters (like GLUT1 deficiency syndrome). Symptoms typically begin in infancy or early childhood and may include severe watery diarrhea, bloating, gas, abdominal cramps, poor weight gain, and failure to thrive. In some forms, the brain may also be affected if glucose cannot be properly transported across the blood-brain barrier, leading to seizures and developmental delays. Treatment depends on the specific type of disorder but generally involves strict dietary modifications, such as removing the offending sugar from the diet. For example, in glucose-galactose malabsorption, a fructose-based formula replaces standard infant formula. In GLUT1 deficiency, a ketogenic diet can provide the brain with an alternative energy source. Early diagnosis and dietary management are essential to prevent serious complications like dehydration, malnutrition, and developmental problems. With proper treatment, many patients can lead relatively normal lives.

Key symptoms:

Severe watery diarrheaBloating and gasAbdominal pain and crampingPoor weight gain or failure to thriveDehydrationNausea and vomitingIrritability in infantsMalnutritionAcidic or explosive stoolsSeizures (in glucose transporter defects)Developmental delays (in some forms)Low blood sugar episodesMuscle weakness or movement problems (in GLUT1 deficiency)Growth delays

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Jul 2024Genetic Carbohydrate Maldigestion as a Model to Study Food Hypersensitivity

Nottingham University Hospitals NHS Trust

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Disorder of carbohydrate absorption and transport.

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

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Clinical Trials

1 recruitingView all trials with filters →
Other1 trial
Genetic Carbohydrate Maldigestion as a Model to Study Food Hypersensitivity
Active
· Sites: Nottingham · Age: 570 yrs

No specialists are currently listed for Disorder of carbohydrate absorption and transport.

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 Disorder of carbohydrate absorption and transport.

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Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific type of carbohydrate absorption or transport disorder does my child have?,Which foods and ingredients do we need to strictly avoid?,Are there any enzyme replacement therapies or medications available for our specific condition?,How will we monitor growth, nutrition, and development over time?,Should other family members be tested for this condition?,What should we do if my child becomes severely dehydrated?,Are there any clinical trials or new treatments being studied for this condition?

Common questions about Disorder of carbohydrate absorption and transport

What is Disorder of carbohydrate absorption and transport?

Disorder of carbohydrate absorption and transport is a group of rare inherited conditions in which the body has trouble absorbing or moving sugars and carbohydrates from the intestines into the bloodstream, or from the blood into the cells that need them. Carbohydrates are a major source of energy, and when they cannot be properly absorbed or transported, it can lead to a wide range of digestive and nutritional problems. This group includes several specific conditions such as glucose-galactose malabsorption, congenital sucrase-isomaltase deficiency, congenital lactase deficiency, and disorder

Are there clinical trials for Disorder of carbohydrate absorption and transport?

Yes — 1 recruiting clinical trial is currently listed for Disorder of carbohydrate absorption and transport on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.