New research from the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN). This summary is based on a paper published in the journal Annals of Clinical and Translational Neurology on January 9, 2026, titled "Characterization of Clinical Phenotype to Glial Fibrillary Acidic Protein Concentrations in Alexander Disease."

Read the paper here. 

Learn more about GLIA-CTN. 

Transcript: 

New research from the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN), a research group of the Rare Diseases Clinical Research Network.

Exploring the Use of Glial Fibrillary Acidic Protein as a Biomarker in Alexander Disease.

This summary is based on a paper published in the journal Annals of Clinical and Translational Neurology on January 9, 2026.

Alexander disease is a rare disorder of the nervous system characterized by leukodystrophy, or the destruction of myelin (the fatty coating surrounding nerve fibers). In patients with Alexander disease, variants in the GFAP gene lead to the buildup of glial fibrillary acidic protein (GFAP) in the body. Not much is known about the relationship between GFAP levels and disease characteristics. 

In this study, researchers explored the use of GFAP as a biomarker in Alexander disease. First, the team collected cerebrospinal fluid and plasma from participants with and without Alexander disease. Next, they compared the concentration of GFAP over time between these groups, including those with common disease characteristics or genetic variants.

Results showed that GFAP increases over time in young children with Alexander disease. The highest concentrations of GFAP were seen in those with the cerebral disease type. Authors note that these findings are a critical initial step in defining biomarker validation and context of use for GFAP in Alexander disease.