One-Patient trial tests hope for devastating rare disease

NCT ID NCT05998395

Summary

This study tested an oral drug called ruxolitinib in a single adult patient with a rare, severe disease that damages blood vessels in the brain and spinal cord. The goal was to see if the drug could stop the disease from getting worse by preventing new brain lesions. The patient took the drug for up to 26 weeks while doctors closely monitored their condition with scans and tests.

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Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.