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.
journal_name
Muscle Nervejournal_title
Muscle & nerveauthors
McDonald CM,Henricson EK,Abresch RT,Florence JM,Eagle M,Gappmaier E,Glanzman AM,PTC124-GD-007-DMD Study Group.,Spiegel R,Barth J,Elfring G,Reha A,Peltz Sdoi
10.1002/mus.23902subject
Has Abstractpub_date
2013-09-01 00:00:00pages
343-56issue
3eissn
0148-639Xissn
1097-4598journal_volume
48pub_type
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