Metal transport or utilization disorder with epilepsy

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ORPHA:225692
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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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What is Metal transport or utilization disorder with epilepsy?

Metal transport or utilization disorder with epilepsy is an extremely rare group of genetic conditions in which the body has trouble moving or using certain metals that are essential for normal brain and body function. Metals like copper, zinc, manganese, and iron play important roles in how our cells work, especially in the brain and nervous system. When the body cannot properly transport or use these metals, it can lead to a buildup or deficiency of specific metals in certain tissues, causing damage over time. The hallmark feature of this group of disorders is epilepsy — recurrent seizures that can vary in type and severity. Patients may also experience developmental delays, intellectual disability, movement problems, and other neurological symptoms. The seizures often begin in infancy or early childhood and may be difficult to control with standard anti-seizure medications. Treatment depends on the specific underlying metal transport defect. In some cases, supplementing the deficient metal or using chelation therapy to remove excess metal can help. Anti-epileptic drugs are used to manage seizures, though response varies. Because these conditions are so rare, management is highly individualized and typically requires a team of specialists including neurologists, metabolic disease experts, and geneticists. Research is ongoing to better understand the specific genetic causes and develop more targeted therapies.

Key symptoms:

Seizures or epilepsyDevelopmental delayIntellectual disabilityPoor muscle tone or floppinessMovement difficulties or abnormal movementsTrouble feeding in infancySlow growth or failure to thriveSpeech and language delaysAbnormal hair or skin changesLiver problemsVision problemsHearing difficultiesIrritability or behavioral changesLoss of previously learned skills

Inheritance
Variable
Can be inherited in different ways depending on the underlying gene
Age of Onset
Infantile
Begins in infancy, roughly 1 month to 2 years old
Orphanet ↗NORD ↗

Treatments

Source: openFDA + DailyMed · NDA / BLA labels with structured indications · refreshed weekly

No FDA-approved treatments are currently listed for Metal transport or utilization disorder with epilepsy.

View clinical trials →

Source: ClinicalTrials.gov · synced daily · phases, status, and PI names normalized at ingest

No actively recruiting trials found for Metal transport or utilization disorder with epilepsy at this time.

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

Search ClinicalTrials.gov ↗Join the Metal transport or utilization disorder with epilepsy community →

Source: NPI Registry + PubMed · trial PI roles cross-referenced with ClinicalTrials.gov · ranked by match score (publications + PI activity + community signal)

No specialists are currently listed for Metal transport or utilization disorder with epilepsy.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers

Source: NORD Rare Disease Centers + NIH Undiagnosed Diseases Network (UDN) · centers verified active within last 12 months

🏨 Children's

Children's Hospital Colorado Rare Disease Program

Children's Hospital Colorado

📍 Aurora, CO

👤 Boston Children's Hospital Rare Disease Program

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Boston Children's Hospital Rare Disease Program

Boston Children's Hospital

📍 Boston, MA

👤 Boston Children's Hospital Rare Disease Program

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

🏨 Children's

Ann & Robert H. Lurie Children's Hospital Genetics

Lurie Children's Hospital

📍 Chicago, IL

👤 Boston Children's Hospital Rare Disease Program

🏥 NORD

Cincinnati Children's Hospital Medical Center

Cincinnati Children's

📍 Cincinnati, OH

👤 Boston Children's Hospital Rare Disease Program

🏨 Children's

Nationwide Children's Hospital Rare Disease Center

Nationwide Children's Hospital

📍 Columbus, OH

👤 Boston Children's Hospital Rare Disease Program

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

Travel Grants

No travel grants are currently matched to Metal transport or utilization disorder with epilepsy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Metal transport or utilization disorder with epilepsy

Source: PubMed + NIH RePORTER + openFDA + clinical-journal RSS · last 30 days · disease-tagged at ingest by AI extraction with human QC

No recent news articles for Metal transport or utilization disorder with epilepsy.

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

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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 specific metal transport defect does my child have, and which gene is affected?,Are there any targeted treatments such as metal supplementation or chelation that could help?,What type of seizures does my child have, and what is the best medication to control them?,How often should blood metal levels and other labs be monitored?,What developmental therapies do you recommend, and how often should they occur?,Are there any clinical trials or experimental treatments available for this condition?,Should other family members be tested for this genetic condition?

Common questions about Metal transport or utilization disorder with epilepsy

What is Metal transport or utilization disorder with epilepsy?

Metal transport or utilization disorder with epilepsy is an extremely rare group of genetic conditions in which the body has trouble moving or using certain metals that are essential for normal brain and body function. Metals like copper, zinc, manganese, and iron play important roles in how our cells work, especially in the brain and nervous system. When the body cannot properly transport or use these metals, it can lead to a buildup or deficiency of specific metals in certain tissues, causing damage over time. The hallmark feature of this group of disorders is epilepsy — recurrent seizures

At what age does Metal transport or utilization disorder with epilepsy typically begin?

Typical onset of Metal transport or utilization disorder with epilepsy is infantile. Age of onset can vary across affected individuals.

Frequently asked questions about Metal transport or utilization disorder with epilepsy

Auto-generated from canonical disease facts (Orphanet, OMIM, ClinicalTrials.gov, openFDA, NPPES). Not a substitute for clinical guidance.

  1. What is Metal transport or utilization disorder with epilepsy?

    Metal transport or utilization disorder with epilepsy is a rare disease catalogued in international rare-disease ontologies (Orphanet ORPHA:225692). It is typically inherited as variable. Age of onset is generally infantile. For verified primary sources, see the UniteRare Metal transport or utilization disorder with epilepsy page.

  2. How is Metal transport or utilization disorder with epilepsy inherited?

    Metal transport or utilization disorder with epilepsy follows variable inheritance. Genetic counseling is recommended for affected families to understand recurrence risk in offspring and the likelihood of unaffected siblings being carriers. Variants in the underlying gene(s) may be identified via clinical genetic testing.

  3. Are there FDA-approved treatments for Metal transport or utilization disorder with epilepsy?

    Approved treatments for Metal transport or utilization disorder with epilepsy are tracked from openFDA and DailyMed primary sources. Many rare diseases have no specific FDA-approved therapy; for those, supportive care and management of complications form the basis of clinical care. Orphan-drug-designation status is noted where applicable.

  4. Are there clinical trials for Metal transport or utilization disorder with epilepsy?

    Active clinical trials for Metal transport or utilization disorder with epilepsy are tracked daily from ClinicalTrials.gov. Trial availability changes frequently; check the UniteRare trial listings for the current count and recruitment status. Sponsors of rare-disease research often welcome inquiries even when a trial is not actively recruiting at a given moment.

  5. How do I find a specialist for Metal transport or utilization disorder with epilepsy?

    Verified Metal transport or utilization disorder with epilepsy specialists are identified through ClinicalTrials.gov principal-investigator records, peer-reviewed publication authorship (via PubMed), and the NPPES NPI registry. NORD-designated Centers of Excellence and NIH-affiliated rare-disease clinics are also tracked. UniteRare's specialist directory is updated continuously as new evidence becomes available.

See full Metal transport or utilization disorder with epilepsy page for complete clinical details, sources, and verified-specialist listings.

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