Aicardi-Goutieres Syndrome (AGS) Research

This study gathers natural history information about your child’s Aicardi-Goutières syndrome disease progression. A visit to CHOP is not required.

Individuals with a confirmed diagnosis of Aicardi-Goutières syndrome (AGS) may qualify to participate in a Phase II clinical trial investigating the safety and efficacy of a Janus kinase (JAK) inhibitor known as baricitinib.

Individuals with a suspected diagnosis of leukodystrophy may be eligible to participate in a research study investigating the efficacy of whole genome sequencing as a first-line diagnostic tool.

The Myelin Disorders Biorepository Project (MDBP) collects and analyzes clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future scientific research.