Tofersen Offers Lasting Benefit for SOD1 Amyotrophic Lateral Sclerosis

TUESDAY, Jan. 6, 2026 (HealthDay News) -- Tofersen shows long-term benefits for patients with SOD1 amyotrophic lateral sclerosis (ALS), according to a study published online Dec. 22 in JAMA Neurology.Timothy M. Miller, M.D., from the Washington University School of Medicine in St. Louis, and colleagues conducted an integrated analysis of a randomized placebo-controlled trial of adults with weakness attributable to ALS and confirmed SOD1 pathogenic variant to compare early-start versus placebo/delayed-start (about six months later) treatment with tofersen. Participants were randomly assigned to receive tofersen (100 mg) or placebo during a 24-week period (108 participants: 36 in the placebo/delayed-start and 72 in the early-start groups) and could then enroll in an open-label extension (OLE; 95 participants enrolled) with tofersen treatment.The researchers found that at OLE completion, participants could have accumulated 3.5 years or more of follow-up from the start of the randomized trial. Over 148 weeks, earlier versus later initiation of tofersen was associated with numerically less decline in measures of clinical function, respiratory function, muscle strength, and quality of life. Relative to the expected natural history of SOD1-ALS, tofersen prolonged survival. Most adverse events were consistent with progression of ALS or known procedural adverse events."Tofersen shows benefits compared with what we expect to see for these participants, with about 25 percent of participants experiencing improvement. These results provide hope that we can change the trajectory of this devastating disease, and we are optimistic we can do the same for other forms of ALS," Miller said in a statement.Several authors disclosed ties to the biopharmaceutical industry, including Biogen, which manufactures tofersen and funded the study.Abstract/Full Text.Sign up for our weekly HealthDay newsletter

Tofersen Offers Lasting Benefit for SOD1 Amyotrophic Lateral Sclerosis

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