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Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study.
Muntoni F, Signorovitch J, Sajeev G, Done N, Yao Z, Goemans N, McDonald C, Mercuri E, Niks EH, Wong B, Vandenborne K, Straub V, de Groot IJM, Tian C, Manzur A, Dieye I, Lane H, Ward SJ, Servais L; PRO-DMD-01 study investigators; Association Française contre les Myopathies; UK NorthStar Clinical Network; ImagingDMD investigators; cTAP. Muntoni F, et al. PLoS One. 2024 Jul 10;19(7):e0304984. doi: 10.1371/journal.pone.0304984. eCollection 2024. PLoS One. 2024. PMID: 38985784 Free PMC article.
Characterization of patients with Duchenne muscular dystrophy across previously developed health states.
Muntoni F, Goemans N, Posner N, Signorovitch J, Johnson M, He C, Dorling P, Beaverson K, Alvir J, Mahn M, Ward SJ, McDonald CM, Mercuri E; PRO-DMD-01 study, the Imaging DMD study, iMDEX, the collaborative Trajectory Analysis Project (cTAP). Muntoni F, et al. PLoS One. 2024 Oct 30;19(10):e0307118. doi: 10.1371/journal.pone.0307118. eCollection 2024. PLoS One. 2024. PMID: 39475941 Free PMC article.
Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials.
McDonald CM, Signorovitch J, Mercuri E, Niks EH, Wong B, Fillbrunn M, Sajeev G, Yim E, Dieye I, Miller D, Ward SJ, Goemans N; Investigators from the PRO-DMD-01 Study, Collaborative Trajectory Analysis Project (cTAP). McDonald CM, et al. PLoS One. 2024 Jun 3;19(6):e0304099. doi: 10.1371/journal.pone.0304099. eCollection 2024. PLoS One. 2024. PMID: 38829874 Free PMC article.
Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collaboration.
Goemans N, Wong B, Van den Hauwe M, Signorovitch J, Sajeev G, Cox D, Landry J, Jenkins M, Dieye I, Yao Z, Hossain I, Ward SJ; Collaborative Trajectory Analysis Project (cTAP). Goemans N, et al. PLoS One. 2020 Jun 18;15(6):e0232870. doi: 10.1371/journal.pone.0232870. eCollection 2020. PLoS One. 2020. PMID: 32555695 Free PMC article. Clinical Trial.
Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls.
Muntoni F, Signorovitch J, Sajeev G, Goemans N, Wong B, Tian C, Mercuri E, Done N, Wong H, Moss J, Yao Z, Ward SJ, Manzur A, Servais L, Niks EH, Straub V, de Groot IJ, McDonald C; North Star Clinical Network, PRO-DMD-01 Study, The Association Française contre les Myopathies (AFM), The DMD Italian Group, and The Collaborative Trajectory Analysis Project (cTAP). Muntoni F, et al. Neuromuscul Disord. 2022 Apr;32(4):271-283. doi: 10.1016/j.nmd.2022.02.009. Epub 2022 Feb 25. Neuromuscul Disord. 2022. PMID: 35396092 Free article.
Corrigendum to "Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy" [Neuromuscular Disorders 26/9 (2016) 576-583].
Mercuri E, Signorovitch JE, Swallow E, Song J, Ward SJ; DMD Italian Group; collaborative Trajectory Analysis Project (cTAP). Mercuri E, et al. Neuromuscul Disord. 2017 May;27(5):e1. doi: 10.1016/j.nmd.2017.01.004. Epub 2017 Mar 8. Neuromuscul Disord. 2017. PMID: 28284874 Free PMC article. No abstract available.