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


Promising Results for Investigational Treatments for Inherited ALS

Two preliminary studies were published simultaneously in the New England Journal of Medicine, both aimed at reducing superoxide dismutase 1 (SOD1) protein levels in people who had amyotrophic lateral sclerosis (ALS) due to SOD1 mutations. In the first study, a phase I/II trial of intrathecal tofersen, Timothy Miller, MD, PhD, of Washington University in St. […]

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

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).