Figure 1.
Inclusion/exclusion criteria for study enrollment.
Figure 2.
Consort flow sheet.
Table 1.
Patient Demographics.
Table 2.
Treatment-related Adverse Events (AE).
Table 3.
Test-retest Reliability of the MHFMS-Extend S1 vs. S2.
Figure 3.
Box Plots of baseline values for Fine Motor Module, Gross Motor Module, and Timed Tests of Function.
The Values on the ordinate refer to seconds for the timed tests, and absolute scores for the motor modules. Δ indicates mean; – indicates median.
Table 4.
Reliability (Test-Related) of Timed Tests and Tests of Motor Function.
Table 5.
Timed Tests and Motor Function; 6 Month Observed Values and Change from Baseline.
Table 6.
Timed Tests and Motor Function 12 Month; Observed Value and Change from Baseline.
Figure 4.
Box plot of baseline myometry data (column 1) and change from baseline (CFB) at 6 months (column 2) and 12 months (column 3).
LE = lower extremity; TE = total myometry; UE = upper extremity. Δ indicates mean; – indicates median.
Table 7.
Change from Baseline (CFB) at 6 and 12 Months in Compound Motor Action Potential (CMAP).
Table 8.
Child Self-assessed PedsQL; Change from Baseline (CFB) at 6 and 12 Month.
Table 9.
Valproic Acid Trough Levels by Visit.
Table 10.
Mean normalized relative amount of SMN mRNA at 6 and 12 Months Actual Values and Change from Baseline (CFB) in full length (fl) and Δ7SMN.