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The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.

  • Author(s): McDonald, Craig M
  • Henricson, Erik K
  • Abresch, R Ted
  • Florence, Julaine M
  • Eagle, Michelle
  • Gappmaier, Eduard
  • Glanzman, Allan M
  • PTC124-GD-007-DMD Study Group
  • Spiegel, Robert
  • Barth, Jay
  • Elfring, Gary
  • Reha, Allen
  • Peltz, Stuart
  • et al.

Published Web Location

https://doi.org/10.1002/mus.23902
Abstract

INTRODUCTION:Duchenne muscular dystrophy (DMD) subjects ≥5 years with nonsense mutations were followed for 48 weeks in a multicenter, randomized, double-blind, placebo-controlled trial of ataluren. Placebo arm data (N = 57) provided insight into the natural history of the 6-minute walk test (6MWT) and other endpoints. METHODS:Evaluations performed every 6 weeks included the 6-minute walk distance (6MWD), timed function tests (TFTs), and quantitative strength using hand-held myometry. RESULTS:Baseline age (≥7 years), 6MWD, and selected TFT performance are strong predictors of decline in ambulation (Δ6MWD) and time to 10% worsening in 6MWD. A baseline 6MWD of <350 meters was associated with greater functional decline, and loss of ambulation was only seen in those with baseline 6MWD <325 meters. Only 1 of 42 (2.3%) subjects able to stand from supine lost ambulation. CONCLUSION:Findings confirm the clinical meaningfulness of the 6MWD as the most accepted primary clinical endpoint in ambulatory DMD trials.

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