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Myoclonus, cerebellar ataxia, deafness syndrome

Last edited: 4/15/2026

Overview

Cerebellar ataxia, deafness, and myoclonus syndrome, often associated with cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL), is a hereditary condition characterized by neurological deficits including cognitive decline, movement disorders, and vascular complications 1.

Diagnosis

  • Genetic Testing: Identification of mutations in the Notch3 gene, particularly novel mutations affecting exon 16, can confirm diagnosis 2.
  • Neuroimaging: MRI showing diffuse hyperintense signals in subcortical white matter and basal ganglia supports the diagnosis 3.
  • Skin Biopsy: Presence of PAS-positive granules and thickened dermal vessels can further validate the diagnosis 3.
  • Management

  • Supportive Care: Focus on managing symptoms such as cognitive decline, movement disorders, and vascular complications through multidisciplinary approaches 1.
  • Preventive Measures: Regular monitoring for cardiovascular risks and management of secondary complications (e.g., venous insufficiency) 2.
  • Special Populations

  • Elderly: Age is a significant predictor of clinical deterioration, necessitating closer monitoring and tailored management strategies 1.
  • Key Recommendations

  • Genetic Testing for Notch3 Mutations: Essential for confirming CADASIL diagnosis (Evidence: Moderate 23).
  • MRI for Subcortical Lesions: Routine neuroimaging to identify characteristic white matter changes (Evidence: Moderate 3).
  • Close Monitoring in Older Patients: Given increased risk of clinical decline with age, elderly patients require more frequent assessments (Evidence: Moderate 1).
  • References

    1 Caeiro L, Ferro JM. Cognitive profile in CADASIL patients. Journal of neurology, neurosurgery, and psychiatry 2006. link 2 Saiki S, Sakai K, Saiki M, Kitagawa Y, Umemori T, Murata K et al.. Varicose veins associated with CADASIL result from a novel mutation in the Notch3 gene. Neurology 2006. link 3 Panagariya A, Sharma B, Shubhakaran. CADASIL in a family from north-west India. The Journal of the Association of Physicians of India 2004. link

    Original source

    1. [1]
      Cognitive profile in CADASIL patients.Caeiro L, Ferro JM Journal of neurology, neurosurgery, and psychiatry (2006)
    2. [2]
      Varicose veins associated with CADASIL result from a novel mutation in the Notch3 gene.Saiki S, Sakai K, Saiki M, Kitagawa Y, Umemori T, Murata K et al. Neurology (2006)
    3. [3]
      CADASIL in a family from north-west India.Panagariya A, Sharma B, Shubhakaran The Journal of the Association of Physicians of India (2004)

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