Timothy Miller, MD, PhD
Washington University
Using the Answer ALS Dataset to Expand Genetically Defined ALS Subtypes
Over the past few decades, researchers have made great strides in understanding how certain genetic mutations can lead to ALS. However, most patients do not present with these mutations, and it is often difficult to predict whether our understanding of and therapeutic approaches to these specific genetic forms of ALS can be generalized to other patients. By leveraging the Answer ALS dataset, this project seeks to better understand how patients with known genetic forms of ALS compare to those without, informing the design of effective clinical trials and possibly expanding the use of existing therapeutics.