HIV drugs show promise for rare childhood brain disorder

NCT ID NCT02363452

First seen Nov 01, 2025 · Last updated Jun 15, 2026 · Updated 38 times

Summary

This study tested whether drugs normally used for HIV could help children with Aicardi-Goutières syndrome (AGS), a rare genetic disease that causes severe brain inflammation. Eleven children received reverse transcriptase inhibitors to see if the drugs could lower abnormal immune signals (interferon levels) in their blood and spinal fluid. The goal was to control the disease, not cure it, since children with AGS need lifelong management.

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Contacts and locations

Locations

  • Hôpital Necker - Enfants Malades

    Paris, 75015, France

Conditions

Explore the condition pages connected to this study.