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

Muscle Nerve. 2013 Sep;48(3):343-56. doi: 10.1002/mus.23902. Epub 2013 Jun 26.


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.

Keywords: 6-minute walk test; Duchenne muscular dystrophy; ambulation; dystrophinopathy; myometry; natural history; prediction of loss of function; timed function tests.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Double-Blind Method
  • Electromyography
  • Exercise Test*
  • Glucocorticoids / therapeutic use
  • Hand Strength / physiology
  • Humans
  • Longitudinal Studies
  • Male
  • Muscular Dystrophy, Duchenne / drug therapy
  • Muscular Dystrophy, Duchenne / physiopathology*
  • Observation
  • Outcome Assessment, Health Care*
  • Oxadiazoles / therapeutic use
  • Predictive Value of Tests
  • Time Factors
  • Walking / physiology*


  • Glucocorticoids
  • Oxadiazoles
  • ataluren