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Table 1.

Patient characteristics at first NSAA assessment.

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Table 1 Expand

Table 2.

Estimates of MCID for NSAA (raw score) and 6MWD based on distribution methods.

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Table 2 Expand

Table 3.

Estimates of MCID for NSAA (linearized scale) based on distribution methods.

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Table 3 Expand

Fig 1.

Scatter plot of annual changes in 6MWD vs NSAA total score (non-overlapping changes).

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Fig 1 Expand

Table 4.

Patient (boys with DMD) characteristics and perceived mobility status.

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Table 4 Expand

Fig 2.

Preventing loss of function.

Distribution of participant responses for significant change as minimum number of items on NSAA for preventing loss of activity (i.e., preventing a change in score from 1 to 0). The x-axis represents minimum number of sub items for which prevention of loss of function was meaningful for a parent (red) and for a patient with DMD (blue).

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Fig 2 Expand

Fig 3.

Maintaining function.

Distribution of participant responses for significant change as minimum number of items on NSAA for maintenance of function (i.e., preventing a change in score from 2 to 1). The x-axis represents minimum number of sub items for which maintaining the activity was meaningful for a parent (red) and for a patient with DMD (blue).

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Fig 3 Expand

Fig 4.

Participant responses for minimum requirements for participating in a clinical trial lasting 2 years based on improvement of motor function, slowing the decline of motor function, or stopping the decline of motor function of items in the NSAA.

NA = missing response.

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Fig 4 Expand