The DOI matches its Crossref record; 68 of the 68 references it cites were found in a registry or an index.
The 6‐minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study
Open accessAbstract
Introduction: An international clinical trial enrolled 174 ambulatory males ≥5 years old with nonsense mutation Duchenne muscular dystrophy (nmDMD). Pretreatment data provide insight into reliability, concurrent validity, and minimal clinically important differences (MCIDs) of the 6‐minute walk test (6MWT) and other endpoints. Methods: Screening and baseline evaluations included the 6‐minute walk distance (6MWD), timed function tests (TFTs), quantitative strength by myometry, the PedsQL, heart rate–determined energy expenditure index, and other exploratory endpoints. Results: The 6MWT proved feasible and reliable in a multicenter context. Concurrent validity with other endpoints was excellent. The MCID for 6MWD was 28.5 and 31.7 meters based on 2 statistical distribution methods. Conclusions: The ratio of MCID to baseline mean is lower for 6MWD than for other endpoints. The 6MWD is an optimal primary endpoint for Duchenne muscular dystrophy (DMD) clinical trials that are focused therapeutically on preservation of ambulation and slowing of disease progression. Muscle Nerve 48: 357–368, 2013
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Cites
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Cited by
Birnkrant, D. J., Bushby, K., Bann, C. M., Apkon, S. D., Blackwell, A., Brumbaugh, D., Case, L. E., Clemens, P. R., Hadjiyannakis, S., Pandya, S., et al. (2018) · The Lancet Neurology · doi:10.1016/s1474-4422(18)30024-3 · Cited by 1,370
McDonald, C. M., Campbell, C., Torricelli, R. E., Finkel, R. S., Flanigan, K. M., Goemans, N., Heydemann, P., Kaminska, A., Kirschner, J., Muntoni, F., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)31611-2 · Cited by 452
Self-citationMendell, J. R., Sahenk, Z., Lehman, K., Nease, C., Lowes, L. P., Miller, N. F., Iammarino, M. A., Alfano, L. N., Nicholl, A., Al-Zaidy, S., et al. (2020) · JAMA Neurology · doi:10.1001/jamaneurol.2020.1484 · Cited by 405
Duchenne and Becker Muscular Dystrophies
Flanigan, K. M. (2014) · Neurologic Clinics · doi:10.1016/j.ncl.2014.05.002 · Cited by 317
Carlier, P. G., Marty, B., Scheidegger, O., Loureiro de Sousa, P., Baudin, P.-Y., Snezhko, E., & Vlodavets, D. (2016) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-160145 · Cited by 186
Metadata
- Authors
- McDonald, C. M., Henricson, E. K., Abresch, R. T., Florence, J., Eagle, M., Gappmaier, E., Glanzman, A. M., PTC124‐GD‐007‐DMD Study Group, Spiegel, R., Barth, J., Elfring, G., Reha, A., & Peltz, S. W.
- Venue
- Muscle & Nerve
- Publisher
- Wiley
- Published
- 2013-07-17
- Volume
- 48
- Issue
- 3
- Pages
- 357-368
- ISSN
- 0148-639X (ISSN-L)
- 1097-4598
- OpenAlex
- W1951817210
- Cited by
- 354
- References
- 68
- FWCI
- 10.06
- Type
- journal article
- Language
- en
- Open access
- bronze
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- McDonald, C. M., Henricson, E. K., Abresch, R. T., Florence, J., Eagle, M., Gappmaier, E., Glanzman, A. M., PTC124‐GD‐007‐DMD Study Group, Spiegel, R., Barth, J., Elfring, G., Reha, A., & Peltz, S. W. (2013). The 6‐minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study. Muscle & Nerve. https://doi.org/10.1002/mus.23905
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