New drug shows promise for rare childhood brain disorder
NCT ID NCT03921554
First seen Jan 07, 2026 · Last updated Apr 30, 2026 · Updated 13 times
Summary
This study tested a drug called baricitinib in 54 people with Aicardi Goutières Syndrome (AGS), a rare genetic condition that causes brain inflammation and developmental problems. The goal was to see if the drug could safely slow or stop the disease's progression. Researchers measured changes in a neurological scale over 52 weeks to assess effectiveness.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Conditions
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