© 2014 Springer Science+Business Media New York. All rights reserved. Alexander disease is a protein aggregation disorder resulting from mutations in the intermediate filament protein, GFAP. Progress in the past 15 years has defined numerous aspects of astrocyte function that are impacted by these mutations, and that might be amenable to correction. Since reversal of astrocyte dysfunction is likely to be valuable in a wide variety of conditions, Alexander disease offers unique opportunities for exploring the newly emerging field of astrotherapeutics.