Targeted Proteomics upon Treatment with Tofersen Identifies Novel Response Markers for Superoxide Dismutase 1-Linked Amyotrophic Lateral Sclerosis.

In contrast, the reduction of NPTX1, NPTX2, and NPTXR showed a highly significant correlation with the clinical response to tofersen (NPTX1: Pearson's correlation = −0.54, p value = 0.00538045, adjusted R 2 = 0.26, 95% CI −0.77, −0.18; NPTX2: Pearson's correlation = −0.64, p value = 0.00059559, adjusted R 2 = 0.38, 95% CI −0.83, −0.33; NPTXR: Pearson's correlation = −0.53, p value = 0.00602491, adjusted R 2 = 0.25, 95% CI −0.77, −0.18; Fig. 6D–F ).

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