AICARDI GOUTIERES SYNDROME
Clinical trials for AICARDI GOUTIERES SYNDROME explained in plain language.
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New hope for children with devastating genetic brain disorder
Disease control CompletedThis study tested whether a drug called baricitinib could help control symptoms and slow disease progression in children with Aicardi Goutières Syndrome (AGS), a rare and severe genetic disorder. Fifty-four participants, including infants and children, took the drug for a year to…
Matched conditions: AICARDI GOUTIERES SYNDROME
Phase: PHASE2 • Sponsor: Adeline Vanderver, MD • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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New gene test cuts diagnosis time for devastating childhood brain diseases
Diagnosis CompletedThis study tested whether using whole genome sequencing (a complete genetic map) as a first test could provide faster and more accurate diagnoses for children with rare, inherited brain disorders called leukodystrophies. Researchers compared the new approach to the standard step-…
Matched conditions: AICARDI GOUTIERES SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Diagnosis
Last updated Apr 03, 2026 14:43 UTC