Comprehensive, easy-to-understand information about this condition
How we create this content →Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Consider asking your healthcare providers these condition-specific questions
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Consider asking your healthcare providers these condition-specific questions
The documentation for cerebral arteriopathy with subcortical infarcts and leukoencephalopathy is limited primarily due to its rarity, which affects fewer than 1 in 100,000 individuals worldwide. This rarity has led to a lack of systematic clinical studies and comprehensive characterization of the disease. The genetic basis was only recently identified, and ongoing clinical characterization is necessary to better understand the condition.
To navigate your care effectively, seek a neurologist with expertise in hereditary cerebrovascular disorders, as they can provide specialized guidance. Additionally, consider reaching out to the CADASIL Eradication Project at https://notch3.org for support and resources. Participating in patient registries or natural history studies can also be beneficial, as these opportunities may provide insights into your condition and connect you with others facing similar challenges.
Currently, there are two orphan drugs designated for this condition: palm tocotrienol complex and a peptide fraction derived from porcine brain protein, both of which are still in development. Additionally, there are 20 active clinical trials exploring various aspects of cerebral arteriopathy with subcortical infarcts and leukoencephalopathy. For more information on these trials, you can visit ClinicalTrials.gov at https://clinicaltrials.gov/search?cond=cerebral%20arteriopathy%20with%20subcortical%20infarcts%20and%20leukoencephalopathy.
Actionable guidance for navigating care for cerebral arteriopathy with subcortical infarcts and leukoencephalopathy
To navigate your care effectively, seek a neurologist with expertise in hereditary cerebrovascular disorders, as they can provide specialized guidance. Additionally, consider reaching out to the CADASIL Eradication Project at https://notch3.org for support and resources. Participating in patient registries or natural history studies can also be beneficial, as these opportunities may provide insights into your condition and connect you with others facing similar challenges.
Consider asking your healthcare providers these condition-specific questions
Connect with organizations supporting the cerebral arteriopathy with subcortical infarcts and leukoencephalopathy community
Helpful links for rare disease information and support
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 8, 2026
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Consider asking your healthcare providers these condition-specific questions
2 peer-reviewed sources from PubMed
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Organizations with approved therapies for this disease
No approved therapies yet
Research is ongoing — 2 companies have orphan drug designations for this disease
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.