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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

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)...

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Autores principales: Mcdonald, Craig M, Henricson, Erik K, Abresch, R Ted, Florence, Julaine M, Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M, Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Blackwell Publishing Ltd 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3824082/
https://www.ncbi.nlm.nih.gov/pubmed/23681930
http://dx.doi.org/10.1002/mus.23902
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author Mcdonald, Craig M
Henricson, Erik K
Abresch, R Ted
Florence, Julaine M
Eagle, Michelle
Gappmaier, Eduard
Glanzman, Allan M
Spiegel, Robert
Barth, Jay
Elfring, Gary
Reha, Allen
Peltz, Stuart
author_facet Mcdonald, Craig M
Henricson, Erik K
Abresch, R Ted
Florence, Julaine M
Eagle, Michelle
Gappmaier, Eduard
Glanzman, Allan M
Spiegel, Robert
Barth, Jay
Elfring, Gary
Reha, Allen
Peltz, Stuart
author_sort Mcdonald, Craig M
collection PubMed
description 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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spelling pubmed-38240822013-11-14 THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY Mcdonald, Craig M Henricson, Erik K Abresch, R Ted Florence, Julaine M Eagle, Michelle Gappmaier, Eduard Glanzman, Allan M Spiegel, Robert Barth, Jay Elfring, Gary Reha, Allen Peltz, Stuart Muscle Nerve Main Articles 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. Blackwell Publishing Ltd 2013-09 2013-06-26 /pmc/articles/PMC3824082/ /pubmed/23681930 http://dx.doi.org/10.1002/mus.23902 Text en Copyright © 2013 Wiley Periodicals, Inc. http://creativecommons.org/licenses/by/2.5/ Re-use of this article is permitted in accordance with the Creative Commons Deed, Attribution 2.5, which does not permit commercial exploitation.
spellingShingle Main Articles
Mcdonald, Craig M
Henricson, Erik K
Abresch, R Ted
Florence, Julaine M
Eagle, Michelle
Gappmaier, Eduard
Glanzman, Allan M
Spiegel, Robert
Barth, Jay
Elfring, Gary
Reha, Allen
Peltz, Stuart
THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY
title THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY
title_full THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY
title_fullStr THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY
title_full_unstemmed THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY
title_short THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY
title_sort 6-minute walk test and other endpoints in duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study
topic Main Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3824082/
https://www.ncbi.nlm.nih.gov/pubmed/23681930
http://dx.doi.org/10.1002/mus.23902
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