Long-Term Effects of Nusinersen Dosing Frequency on Adult Patients With Spinal Muscular Atrophy: Efficacy of a 6-Month Dosing Interval.

Furthermore, although the mean change in RULM scores was not statistically significant, it showed a trend toward improvement (Table 3 , Figure 2b ), which was similar to that observed in the European cohort.

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In contrast, for the nonambulatory group, the changes in HFMSE scores from baseline at 15, 27, and 39 months were negative (−0.3, −1.4, and −1.3 points, respectively), although these changes did not reach statistical significance (Table 2 ).

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