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


Healey ALS Platform Trial receives “May Proceed” notice for three drugs

The Sean M. Healey & AMG Center for ALS at Mass General has received approval from the U.S. Food and Drug Administration (FDA) to proceed with administering three proposed drug regimens in the HEALEY ALS Platform Trial – the first trial of its kind for amyotrophic lateral sclerosis (ALS).

Miller receives international innovation prize

Recognized for developing experimental treatment for ALS Timothy Miller, MD, PhD, the David Clayson Professor of Neurology at Washington University School of Medicine in St. Louis, and a group of his colleagues have received the inaugural Healey Center International Prize for innovation in amyotrophic lateral sclerosis (ALS) research from the Sean M. Healey & AMG […]

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 population, this […]

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

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).

Scientists Reverse Alzheimer’s-Like Symptoms in Mice

Scientists Reverse Alzheimer’s-Like Symptoms in Mice
Engadget states, “Scientists have discovered a way of counteracting proteins that cause Alzheimer’s-like symptoms including memory loss. Researchers at Washington University in St. Louis, Missouri focus on tau protein tangles.” in an article about Dr. Miller’s 2017 STM publication.

Scientists Stop and Reverse Alzheimer’s-Related Brain Damage in Mice

A news release by MNT examines Dr. Miller’s 2017 STM publication. “The team studied the effects of the synthetic molecules on crab-eating macaques. They administered two doses of antisense oligonucleotides or two doses of a placebo, 1 week apart, directly into the cerebrospinal fluid – as one would when treating humans. The trial revealed that, in monkeys […]

Molecule Throws Roadblock in Alzheimer’s Path

Molecule Throws Roadblock in Alzheimer’s Path
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 […]