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

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.

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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.

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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 […]


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