Doctors repurpose HIV drugs in bid to calm rare child brain disease

NCT ID NCT02363452

Summary

This study tested whether three medications commonly used to treat HIV could help children with Aicardi-Goutières syndrome (AGS), a rare genetic disorder that causes brain inflammation. Eleven children received the drug combination for one year to see if it could reduce the overactive immune response that damages their nervous system. The main goal was to measure changes in a specific immune system marker, not to cure the disease, as ongoing treatment would likely be needed.

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Contacts and locations

Locations

  • Hôpital Necker - Enfants Malades

    Paris, 75015, France

Conditions

Explore the condition pages connected to this study.