Published in
Wiley, Muscle & Nerve, 3(48), p. 343-356, 2013
DOI: 10.1002/mus.23902
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- ORCID
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- [www.ncbi.nlm.nih.gov] | PDF
- [europepmc.org] | PDF
- [www.researchgate.net] | PDF
- [www.ncbi.nlm.nih.gov] | PDF
- [www.ncbi.nlm.nih.gov] | PDF
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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
,
R. Ted Abresch,
Craig M. Mcdonald ,
Henricson Ek,
Erik K. Henricson,
Abresch Rt,
R. Ted Abresch,
Florence Jm,
Julaine M. Florence,
Michelle Eagle,
Eduard Gappmaier,
Glanzman Am,
Allan M. Glanzman,
Study Group Ptc124 Gd 007 Dmd,
Robert Spiegel
and other authors.
Full text: Download
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