Abstract

the most stable group between the two evaluations, in contrast to groups NL and FD, whose gait parameters showed a moderate-tolarge responsiveness. Last column reports values for thewhole CMT group. Discussion and conclusions:At 12-month follow-up, some gait parameters showed higher responsiveness than clinical scales for CMT patients [1,2]. Different parameters showed a better sensitivity to the progression of the disease depending on the degree of locomotor impairment (NL, FD, FD&POD), possibly because at different CMT stages the worsening of locomotor function is related to different biomechanical factors. For example, for NL subjects a complex task such as SD, which requires a greater capacity of coordination and balance, is able to evidencemotor deteriorationmore than level walking. Demanding tasks, such as star descending, toe and heel walking, highlight the functional changes related to disease progression even at the early and intermediate CMT stage. At a more advanced stage, responsiveness of specific gait parameters decreased, likely because of a floor effect, and stride length remained the only one still moderately sensitive to disease progression. The results of this study suggest that stage-specific gait parameters can be used as responsive outcomemeasures to assess the progression of CMT disease and/or the effect of possible drug therapies.

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