Advancing knowledge of human development, developmental disabilities, and neurodegenerative diseases.
The FDA approved Zanvastro, the first drug to treat Alexander Disease, a breakthrough that wouldn’t have been possible without research at the Waisman Center. By Addison Dama for The Daily Cardinal October 8, 2026 | 2:00am CDT The Food and Drug Administration (FDA) approved Zanvastro, the first injectable treatment for Alexander Disease, on Sept. 3. …
Alexander disease (AxD) is a rare neurological disorder caused by dominant gain-of-function mutations in the gene for glial acidic fibrillary protein (GFAP).
New research from Waisman investigator, Tracy Hagemann, PhD, associate research professor, delves into the cognitive impairments and associated molecular changes caused by Alexander disease (AxD), revealing similarities to other neurodegenerative diseases like Alzheimer’s.
Title: Congruent gene expression in Alexander disease model mice and human Alzheimer’s disease Legend: (A) A Rank-Rank Hypergeometric Overlap (RRHO) heatmap comparing a composite gene expression portrait of human Alzheimer’s disease (AD, X axis) with transcriptomic changes in hippocampus from Alexander disease model mice (GFAP mutation, Y axis) indicates high matching of up- and downregulated …
Alexander disease (AxD) is caused by mutations in the gene for glial fibrillary acidic protein (GFAP), an intermediate filament expressed by astrocytes in the central nervous system.
The hallmarks of Alexander disease, aggregation of misfolded GFAP proteins and dysregulation of brain cells called astrocytes, may be stopped and reversed in rodent models with the inactivation of the transcription factor STAT3.
Messing wanted to study if the overexpression of GFAP resulted in a certain reactive response in the brain.
Anastasis is a recently described process in which cells recover after late-stage apoptosis activation. The functional consequences of anastasis for cells and tissues are not clearly understood.
Alexander disease (AxD) is a devastating leukodystrophy caused by gain-of-function mutations in GFAP, and the only available treatments are supportive. Recent advances in antisense oligonucleotide (ASO) therapy have demonstrated that transcript targeting can be a successful strategy for human neurodegenerative diseases amenable to this approach.
