The Miller Laboratory is dedicated to understanding neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and dementias in order to develop new, effective, and safe treatments. We focus on translational neuroscience, new therapeutic approaches for neurodegenerative diseases, and precision medicine.

ALS Research

ALS Research

The Miller Lab seeks to understand the pathogenesis of ALS with basic studies focused on SOD1, C9ORF72, TBK-1,  miRNA, protein misfolding, protein kinetics, and genetic modifiers of disease. We employ RNA-targeted therapeutic strategies, mainly using antisense oligonucleotides.

Learn More

Dementia Research

Dementia Research

The Miller Lab studies how the various isoforms of the protein tau affect neurodegeneration. We employ RNA-targeted therapeutic strategies using antisense oligonucleotides to develop therapies for dementias involving the tau protein as well as for TREM-2.

Learn More

More Translational Research News

Making a Difference

This FDA decision means that people with SOD1 ALS now have access to a treatment that appears to slow down the neurodegenerative disease process and helps preserve motor function. This gives me hope that this could have a positive impact on the quality of life for people living with this rare form of ALS.”

Timothy M. Miller, MD, PhD
A new drug has been approved by the Food and Drug Administration (FDA) for a rare, inherited form of amyotrophic lateral sclerosis (ALS). Called tofersen, the drug — developed by Biogen Inc. and based in part on research conducted at Washington University School of Medicine in St. Louis — slows the progression of the deadly, paralyzing disease.