Seizures-intellectual disability due to hydroxylysinuria syndrome

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:79156OMIM:236900E72.3
Who is this for?
Show terms as
8Treatment centers

Where are you in your journey?

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

Overview

Seizures-intellectual disability due to hydroxylysinuria syndrome (also sometimes called hydroxylysinuria) is an extremely rare inherited metabolic disorder. It affects how the body breaks down a building block of protein called hydroxylysine. When this process does not work properly, hydroxylysine builds up in the blood and spills into the urine. This buildup appears to interfere with normal brain development and function. The main features of this condition include recurring seizures (epilepsy) and intellectual disability, meaning that learning, memory, and daily problem-solving are harder than usual. Some people may also have delays in reaching developmental milestones like talking or walking. The exact reason why too much hydroxylysine harms the brain is not fully understood, and research into this condition is still very limited because so few cases have ever been reported. Treatment is focused on managing symptoms rather than fixing the underlying cause. Seizures are typically treated with anti-epileptic medications. There is currently no cure, and no specific diet or drug has been proven to lower hydroxylysine levels effectively. People with this condition benefit from a team of specialists including neurologists, metabolic geneticists, and developmental therapists who work together to support quality of life.

Key symptoms:

Recurring seizures (epilepsy)Intellectual disability (difficulty with learning and thinking)Delayed speech and language developmentDelayed motor milestones such as sitting or walkingElevated hydroxylysine levels in the urineElevated hydroxylysine levels in the bloodBehavioral difficulties

Clinical phenotype terms (3)— hover any for plain English
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 Seizures-intellectual disability due to hydroxylysinuria syndrome.

View clinical trials →

No actively recruiting trials found for Seizures-intellectual disability due to hydroxylysinuria syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Seizures-intellectual disability due to hydroxylysinuria syndrome community →

No specialists are currently listed for Seizures-intellectual disability due to hydroxylysinuria syndrome.

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 Seizures-intellectual disability due to hydroxylysinuria syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Seizures-intellectual disability due to hydroxylysinuria syndromeForum →

No community posts yet. Be the first to share your experience with Seizures-intellectual disability due to hydroxylysinuria syndrome.

Start the conversation →

Latest news about Seizures-intellectual disability due to hydroxylysinuria syndrome

No recent news articles for Seizures-intellectual disability due to hydroxylysinuria syndrome.

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 genetic testing should we do to confirm the diagnosis and identify the gene involved?,Which anti-epileptic medication is the best starting choice for my child's type of seizures?,What should we do if a seizure lasts longer than 5 minutes — do we need a rescue medication at home?,Are there any dietary changes or supplements that might help manage hydroxylysine levels?,What therapies (speech, occupational, physical) should we start, and how soon?,Are there any research studies or patient registries we can join to help advance understanding of this condition?,What signs of worsening should prompt us to seek urgent medical attention?

Common questions about Seizures-intellectual disability due to hydroxylysinuria syndrome

What is Seizures-intellectual disability due to hydroxylysinuria syndrome?

Seizures-intellectual disability due to hydroxylysinuria syndrome (also sometimes called hydroxylysinuria) is an extremely rare inherited metabolic disorder. It affects how the body breaks down a building block of protein called hydroxylysine. When this process does not work properly, hydroxylysine builds up in the blood and spills into the urine. This buildup appears to interfere with normal brain development and function. The main features of this condition include recurring seizures (epilepsy) and intellectual disability, meaning that learning, memory, and daily problem-solving are harder

How is Seizures-intellectual disability due to hydroxylysinuria syndrome inherited?

Seizures-intellectual disability due to hydroxylysinuria syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Seizures-intellectual disability due to hydroxylysinuria syndrome typically begin?

Typical onset of Seizures-intellectual disability due to hydroxylysinuria syndrome is infantile. Age of onset can vary across affected individuals.