"I have had the opportunity to interact with the ALS advocacy community recently, and I think it is
fair to say, they are frustrated; frustrated with the lack of progress in that refractory and terrible
disease. And when I look at that problem, I see a critical issue—perhaps THE critical issue—a lack of
basic biologic understanding of the disease."
- Norman E. "Ned" Sharpless, MD
Acting Commissioner of the FDA
Filling A Key Knowledge Gap
Researchers still do not fully understand the mechanisms responsible for the onset and progression
of ALS. This knowledge gap presents a significant challenge for ALS drug discovery. Translational
research conducted at ALS TDI is focused on this critical area with the goal of rapidly and efficiently
translating new information into tangible drug discovery methods and high quality drug candidates.
By learning about ALS from people with ALS, we hope to bridge the existing knowledge gap and learn more about the molecular and cellular dysfunction involved in ALS.