Hope for a devastating childhood disease: drug trial aims to halt progression
NCT ID NCT03921554
Summary
This study tested a drug called baricitinib for children with Aicardi Goutières Syndrome (AGS), a rare and severe genetic disorder that causes brain damage and affects many organs. The goal was to see if the drug, taken for a year, could safely reduce harmful inflammation and help stabilize or improve children's neurological function and development. It involved 54 participants, including infants as young as one month old.
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Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Conditions
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