AICARDI GOUTIERES SYNDROME
Clinical trials for AICARDI GOUTIERES SYNDROME explained in plain language.
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Could a JAK inhibitor stabilize rare brain disease?
Disease control CompletedThis study tested the drug baricitinib in 54 people with Aicardi-Goutières Syndrome (AGS), a rare genetic disorder that causes brain inflammation and developmental problems. The goal was to see if the drug could safely stabilize or improve neurologic function over one year. Baric…
Matched conditions: AICARDI GOUTIERES SYNDROME
Phase: PHASE2 • Sponsor: Adeline Vanderver, MD • Aim: Disease control
Last updated May 22, 2026 13:52 UTC
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Gene sequencing breakthrough may unlock mysterious brain diseases in kids
Knowledge-focused CompletedThis study looked at whether whole genome sequencing (a complete scan of a person's DNA) can help doctors diagnose leukodystrophies—rare brain disorders that damage white matter—more accurately than standard methods. About 236 children with abnormal brain MRI scans but no known g…
Matched conditions: AICARDI GOUTIERES SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 18, 2026 12:01 UTC