Figure 2.

Changes in RULM and HFMSE scores during the first 10 months of treatment with Nusinersen. Absolute values of the (top) RULM score (total range 0–37 points, higher indicates better) and the (bottom) HFMSE score (total range 0–66 points, higher indicates better) as well as their changes between baseline and 10th month are depicted for each individual patient. Patients are briefly characterized in brackets: sex, age in years at baseline, type of SMA. RULM (top) and HFMSE (bottom) scores showed clinically meaningful changes in three patients (patient 5, 6, 7), with RULM score depicting an increase before HFMSE score (2 versus 10 months after initiation of the treatment). Patient 3, who is ambulatory, already started with a RULM score of 37 points (maximum score) at baseline. Patient 4 discontinued treatment before the fifth application (and herewith 6th month assessment) of Nusinersen due to the development of a sacral pressure ulcer.

HFMSE, Hammersmith Functional Motor Scale Expanded; F, female; M, male; RULM, Revised Upper Limb Module; SMA, spinal muscular atrophy.