Genetic neurovascular malformation

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ORPHA:371436
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4Specialists8Treatment centers

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

Genetic neurovascular malformation is a rare condition in which abnormal connections form between blood vessels in or around the brain and nervous system due to changes in a person's genes. These malformations can involve arteries, veins, or capillaries and may disrupt normal blood flow in the brain or spinal cord. Depending on the location and size of the malformation, symptoms can range from headaches and seizures to more serious problems like bleeding (hemorrhage) in the brain, stroke-like episodes, or neurological deficits such as weakness or difficulty speaking. This group of conditions is inherited, meaning the genetic changes responsible can be passed from parent to child. Several different genes have been identified that can cause neurovascular malformations, and the specific gene involved often determines the type and severity of the condition. Some people may have visible skin changes or other signs outside the nervous system. Treatment depends on the type, size, and location of the malformation. Options may include monitoring with regular imaging, surgical removal, endovascular procedures (where doctors work inside the blood vessels using catheters), or focused radiation therapy (stereotactic radiosurgery). Medications may be used to manage symptoms like seizures or headaches. Research is ongoing into targeted drug therapies that address the underlying genetic pathways. Because this is a complex condition, care is best managed by a team of specialists working together.

Key symptoms:

Headaches, sometimes severe or suddenSeizuresBleeding in the brain (intracranial hemorrhage)Stroke-like episodesWeakness on one side of the bodyNumbness or tinglingDifficulty speaking or understanding speechVision problemsDizziness or balance problemsRinging in the ears or pulsating sound in the headLearning difficulties or cognitive changesSkin changes such as red or purple birthmarksFatigueNausea or vomiting

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 ↗

Treatments

No FDA-approved treatments are currently listed for Genetic neurovascular malformation.

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No actively recruiting trials found for Genetic neurovascular malformation at this time.

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Search ClinicalTrials.gov ↗Join the Genetic neurovascular malformation community →

Specialists

4 foundView all specialists →
DH
David Ho
Specialist
PI on 2 active trials26 Genetic neurovascular malformation publications
EM
Enrico Novelli, MD
PITTSBURGH, PA
Specialist
PI on 2 active trials

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 Genetic neurovascular malformation.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Genetic neurovascular malformation

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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 specific type of neurovascular malformation does my family member or I have, and which gene is involved?,What is the risk of bleeding or other serious complications?,How often should imaging or monitoring be done?,What treatment options are available, and what are the risks and benefits of each?,Are there any activities or medications I should avoid?,Should other family members be tested for this genetic condition?,Are there any clinical trials or new therapies I should know about?

Common questions about Genetic neurovascular malformation

What is Genetic neurovascular malformation?

Genetic neurovascular malformation is a rare condition in which abnormal connections form between blood vessels in or around the brain and nervous system due to changes in a person's genes. These malformations can involve arteries, veins, or capillaries and may disrupt normal blood flow in the brain or spinal cord. Depending on the location and size of the malformation, symptoms can range from headaches and seizures to more serious problems like bleeding (hemorrhage) in the brain, stroke-like episodes, or neurological deficits such as weakness or difficulty speaking. This group of conditions

Which specialists treat Genetic neurovascular malformation?

4 specialists and care centers treating Genetic neurovascular malformation are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.