Experts across multiple fields are working together to build a better understanding of Alexander Disease and develop treatments with the goal of improving the lives of Patients and their Families.

Current research is aimed at understanding the mechanisms by which the mutations cause disease, developing better animal models for the disorder, and exploring potential strategies for treatment.

At present, there is no exact animal model for the disease; however, mice and rats have been engineered to produce the same mutant forms of GFAP found in individuals with Alexander disease. Cell models are also used to model various aspects of the disease.

One clinical study is underway to identify biomarkers of disease severity or progression in samples of blood or cerebrospinal fluid. Such biomarkers, if found, would be a major advantage for evaluating the response to any treatments that are developed in the future.

Dr Albee Messing has created a number of resources for everyone who wants to learn more about Alexander Disease Science.