Finding clinical meaning in patient-reported functional health: development of the Duchenne muscular dystrophy lifetime mobility scale

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Person-reported outcome (PRO) measures for DMD clinical trials should be tied to meaningful disease milestones such as loss of ability to stand, rise from a chair, climb stairs, walk independently, raise a hand to the mouth, as well as need for non-invasive ventilation. We developed the DMD lifetime mobility scale (DMD-LMS) with data from the CINRG Duchenne natural history study, using mobility items from the PODCI, PedsQL, PedsQL NMM, pediatric and adult mobility scales of the NeuroQOL. “Successful” question items: a) differentiated between milestone groups; b) showed significant change over one-year, and c) were deemed “clinically significant” by exceeding change of 1/3 the standard deviation of the measure.

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