Figure 2

Median pre-post-comparisons to baseline (Δ) at assessed time points for total MRC % and subdomains during nusinersen therapy. Error bars indicate the interquartile range. (a,b) Individual trajectories of assessed patients for non-ambulatory (a) and ambulatory (b) patients showed a marked variability of treatment responses. The n-number indicates the number of analysed individuals. Patients’ pseudonyms are listed alongside the graphs (e.g. S1). (c–f) Depiction of MRC % changes in subdomains over time in ambulatory vs non-ambulatory patients and the overall cohort. Numbers in brackets indicate the number of muscle functions included in that specific subdomain. Improvements in both patient subgroups were observed in the proximal muscles of the upper limb (c). Muscle strength showed no relevant changes in the lower limbs with temporary increases above the threshold of non-ambulatory patients shortly after the loading dosing (d,f) regressing during further therapy. At month two distal muscle strength of ambulatory patients worsened (e,f), but the initial decline lessened over time.