From Vision to Validation: The Journey of Tofersen in Superoxide Dismutase 1 Gene–Associated Amyotrophic Lateral Sclerosis

Few stories in modern neurology illustrate the power of imagination and scientific innovation as the development of tofersen for superoxide dismutase 1 gene (SOD1)-associated amyotrophic lateral sclerosis (ALS). As an ALS clinician-scientist privileged to have cared for patients through the development period, the experience has been elating, even if it has not been without loss. Today, as real-world evidence accumulates from national cohorts and international treatment programs, the story of tofersen is told in a new and perhaps even more consequential chapter, helping a growing number of people.