Dr. Miller’s exciting research in the field of neurodegenerative diseases consistently generates national and international excitement.
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).
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 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 […]
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.
With funds raised through the ALS Ice Bucket Challenge, The ALS Association has been able to invest significantly in the identification of biological indicators (or biomarkers) for ALS.
A genetic therapy that increases or lowers levels of a protein raises hopes for a treatment for neurological disorders.
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).
(CNN) An experimental treatment for the rapidly progressive disease ALS, or amyotrophic lateral sclerosis, has been called potentially "game-changing."
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.
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 […]
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 […]
An article by Scientific American describes Dr. Miller’s Science Translational Medicine publication. “The small molecules cleared and prevented tau buildup in mice and monkeys.”