AICARDI GOUTIERES SYNDROME
Clinical trials for AICARDI GOUTIERES SYNDROME explained in plain language.
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New hope for rare brain disorder: drug targets immune overdrive
Disease control CompletedThis study tested a drug called 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 is safe and can help control symptoms by reducing the immune system's…
Matched conditions: AICARDI GOUTIERES SYNDROME
Phase: PHASE2 • Sponsor: Adeline Vanderver, MD • Aim: Disease control
Last updated May 17, 2026 05:49 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 17, 2026 05:37 UTC