Dr. Miller’s exciting research in the field of neurodegenerative diseases consistently generates national and international interest. 


Investigational drug for genetic form of ALS improves disease’s molecular signs (Links to an external site)

The trial, sponsored by the pharmaceutical company Biogen, showed that an investigational drug, known as tofersen, reduced molecular signs of the fatal disease, but at six months did not improve motor control and muscle strength. However, Miller and colleagues found evidence that longer-term use of the drug may help stabilize muscle strength and control.

Tofersen Phase 3 Drug Results and Use with ALS

At the American Neurological Association (ANA) Annual Meeting, Dr. Timothy Miller presented VALOR study of Tofersen Phase 3 Drug Results and Use with ALS. Tofersen is an antisense drug being evaluated for the potential treatment of SOD1-ALS. For more information and to learn about Tofersen Phase 3 Drug Results please see links below. Biogen News Release; […]

Prospective natural history study of C9orf72ALS clinical characteristics and biomarkers

Prospective natural history study of C9orf72ALS clinical characteristics and biomarkers Our team’s hard work has been published in Neurology. This paper profiles the clinical features, such as age at disease onset, survival duration, and measures of disease progression, of ALS patients with mutations in the C9orf72 gene. By defining the natural history of this patient […]

New Diagnostic Test for Neurofilament

Biomarkers are measures reflective of biological processes that occur in the body. In the setting of disease, biomarkers may be used for diagnostic, prognostic or treatment monitoring purposes.

Experimental Drug Shows Promise for Genetic Form of ALS (Links to an external site)

An early stage trial of an investigational therapy for amyotrophic lateral sclerosis (ALS) suggests that people could tolerate the experimental drug and, in exploratory results, the experimental drug was linked to possible slower progression in people with a genetic form of the disease caused by mutations in a gene called superoxide dismutase 1 (SOD1).

Molecule Throws Roadblock in Alzheimer’s Path (Links to an external site)

An article by New Atlas describing Dr. Miller’s STM work states, “When it comes to the drama of Alzheimer’s disease, the compound in the starring role as the villain is tau. It’s a protein that normally helps our neurons function properly, but one that can clump together into plaques that damage brain cells. Now researchers have […]

Publication by Mariah L. Hoye, PhD Candidate

Congratulations to Miller Lab member Mariah L. Hoye on her publication! Mariah is a PhD candidate who is studying microRNAs, which are small compounds that regulate a variety of processes in the body, and their role in ALS disease progression. Her manuscript that investigates microRNAs specific to motor neurons in ALS rodent models was recently […]

Publication by Kathleen Schoch, PhD

Congratulations to Miller Lab member Kathleen Schoch on her publication! Dr. Kathleen Schoch is a postdoctoral research assistant in the Miller Lab who focuses on dementias characterized by the pathological accumulation of the protein “tau”. She published a paper in Neuron that examined the potential of a unique tau-lowering therapeutic strategy to treat Alzheimer’s disease, […]

Dr. Timothy Miller Wins Sheila Essey Award for ALS Research (Links to an external site)

The ALS Association, in partnership with the AAN and the American Brain Foundation, are awarding research funding to Timothy M. Miller, M.D., Ph.D., the David Clayson Professor of Neurology from the Washington University School of Medicine in St. Louis. The award recognizes significant research contributions in the search for the causes, prevention, and cure for amyotrophic lateral sclerosis (ALS). Since 1996, The ALS Association and the American Academy of Neurology have jointly chosen recipients of the award.

Ste. Genevieve Riverdogs Baseball Team Supports ALS Research

Prior to the final game of the season, the Ste. Genevieve Riverdogs baseball team surprised Coach Jeremy Hoog with a touching tribute as well as a donation to Dr. Timothy Miller’s ALS Research Lab in honor of Coach Hoog’s mother – and the Riverdogs’ biggest fan – Cheryl.