🧠 Neurological Disorder

Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy

Also known as: CADASIL, Hereditary multi-infarct dementia, NOTCH3-related CADASIL

CADASIL is a hereditary stroke disorder caused by mutations in the NOTCH3 gene. It leads to thickening of blood vessel walls, reducing blood flow to the brain and causing a variety of neurological symptoms, including migraine with aura, recurrent stroke, cognitive decline, and psychiatric disturbances. The condition typically manifests in adulthood and progresses over time.

👥 1-9 / 100,000 Prevalence
🔬 15 Active Trials

CADASIL is like having tiny problems in the pipes that bring blood to your brain. These problems can cause headaches, make it hard to think clearly, and sometimes cause strokes. It's something you can get from your parents, and doctors can help manage the symptoms.

Signs & Symptoms

  • Migraine with aura
  • Recurrent stroke or transient ischemic attacks (TIAs)
  • Cognitive decline leading to dementia
  • Psychiatric disturbances (depression, apathy, psychosis)
  • Seizures
  • White matter lesions on brain MRI
  • Muscle weakness
  • Dysarthria (difficulty speaking)
  • Dysphagia (difficulty swallowing)

Treatment Options

MEDICATION FDA Approved

Symptomatic treatment for migraine

MODERATELY EFFECTIVE
MEDICATION FDA Approved

Antiplatelet agents (e.g., aspirin, clopidogrel)

MODERATELY EFFECTIVE
MEDICATION FDA Approved

Antihypertensive medications

MODERATELY EFFECTIVE
THERAPY

Physical therapy

SUPPORTIVE
THERAPY

Occupational therapy

SUPPORTIVE
THERAPY

Speech therapy

SUPPORTIVE
MEDICATION FDA Approved

Psychiatric support and medications for mood disorders

MODERATELY EFFECTIVE

Diagnosis

  • Clinical evaluation
  • Brain MRI (magnetic resonance imaging) showing characteristic white matter lesions
  • Genetic testing for NOTCH3 mutations
  • Skin biopsy with granular osmiophilic material (GOM) deposition in blood vessels

History

CADASIL was first described in 1977 by Marie-Germaine Bousser and colleagues in a French family. The genetic basis of the disease, mutations in the NOTCH3 gene, was identified in 1996 by Elisabeth Tournier-Lasserve's group.

Recent Breakthroughs

2022

Novel therapeutic targets identified in CADASIL pathogenesis

Research has identified potential therapeutic targets by elucidating the role of specific inflammatory pathways and vascular remodeling processes in CADASIL. These findings may lead to the development of targeted therapies to slow disease progression.

2023

Improved MRI techniques for early CADASIL diagnosis

Advanced MRI techniques, such as diffusion tensor imaging (DTI) and quantitative susceptibility mapping (QSM), are being used to detect subtle changes in brain microstructure in early stages of CADASIL, potentially improving diagnostic accuracy and enabling earlier intervention.