Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a "real-world" setting.

Objective: Patients with amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 (SOD1) gene mutations (SOD1 ALS) treated with tofersen have shown slowing of disease progression, and disease stabilization with recovery of function in some patients. We report our clinical experience with treating patients with SOD1 ALS and the effects of tofersen on outcome measures.

Methods: This was a single-center observational study of patients with SOD1 ALS receiving treatment with tofersen.