A highly inter disciplinary, collaborative approach to research centered in partnership with patients and families.

Research Focus

Click on each puzzle piece to see more

Our focus on advancing ALS therapy development has led us to investigate a broad range of topics. Pairing of deep phenotypic data with genetic and environmental exposure data affords an opportunity to identify causes and modifiers of disease. In parallel, we have pioneered novel approaches to data collection and led efforts to discover and validate biomarkers that are fit-for-purpose in both symptomatic and pre-symptomatic ALS. Progress made in these areas has, in turn, fueled our efforts to modify disease course and perhaps even prevent ALS through innovative clinical trials. To learn more, click on each piece of the puzzle below.

Deep Phenotyping

  • Motor
  • Cognitive
  • Phenotype-Genotype

Causes & Modifiers

  • Genetics
  • Environment
  • Gene x Environment

Biomarker Discovery & Validation

  • Biofluid
  • Imaging
  • Electrical
  • Physiological

Novel Data Collection Methods

  • Remote assessments
  • EHR-based

Disease Biology & Drug Discovery

  • iPSC models
  • Drug screening

Clinical Trials

  • Disease modifying
  • Disease prevention

Participate in Research

Collaborative and translational research to find and fill the gaps in our knowledge about the causes and treatment of ALS

Progress towards a cure for ALS is critically dependent on your active participation in a research study. We welcome your interest and willingness to enroll in one of our studies, and we have open opportunities whether you have ALS or a related disorder, are at elevated (genetic) risk for ALS, or are healthy but willing to sign up as a control subject. See below for details of ongoing studies and information as to how you can get involved.

Four Research Vignettes

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