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Page 1
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. Among authors: sajeev g. 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.
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials.
McDonald CM, Sajeev G, Yao Z, McDonnell E, Elfring G, Souza M, Peltz SW, Darras BT, Shieh PB, Cox DA, Landry J, Signorovitch J; ACT DMD Study Group and the Tadalafil DMD Study Group. McDonald CM, et al. Among authors: sajeev g. Muscle Nerve. 2020 Jan;61(1):26-35. doi: 10.1002/mus.26736. Epub 2019 Nov 7. Muscle Nerve. 2020. PMID: 31599456 Free PMC article.
Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord Blood.
Lin C, Sajeev G, Stiff PJ, Brunstein CG, Cutler C, Sanz G, Lindemans CA, Rezvani AR, Hanna R, Koh LP, Maziarz RT, Hwang WYK, Song Y, Liu Q, Manghani R, Sivaraman S, Signorovitch J, Horwitz ME, Sung AD. Lin C, et al. Among authors: sajeev g. Transplant Cell Ther. 2023 Jan;29(1):52.e1-52.e9. doi: 10.1016/j.jtct.2022.09.018. Epub 2022 Sep 28. Transplant Cell Ther. 2023. PMID: 36179986 Free PMC article. Clinical Trial.
Genotype and Lifetime Burden of Disease in Hypertrophic Cardiomyopathy: Insights from the Sarcomeric Human Cardiomyopathy Registry (SHaRe).
Ho CY, Day SM, Ashley EA, Michels M, Pereira AC, Jacoby D, Cirino AL, Fox JC, Lakdawala NK, Ware JS, Caleshu CA, Helms AS, Colan SD, Girolami F, Cecchi F, Seidman CE, Sajeev G, Signorovitch J, Green EM, Olivotto I. Ho CY, et al. Among authors: sajeev g. Circulation. 2018 Oct 2;138(14):1387-1398. doi: 10.1161/CIRCULATIONAHA.117.033200. Epub 2018 Aug 23. Circulation. 2018. PMID: 30297972 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. Among authors: sajeev g. 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.
Suitability of external controls for drug evaluation in Duchenne muscular dystrophy.
Goemans N, Signorovitch J, Sajeev G, Yao Z, Gordish-Dressman H, McDonald CM, Vandenborne K, Miller D, Ward SJ, Mercuri E; investigators from PRO-DMD-01 Study, CINRG DNHS, ImagingDMD, and the DMD Italian Group. Goemans N, et al. Among authors: sajeev g. Neurology. 2020 Sep 8;95(10):e1381-e1391. doi: 10.1212/WNL.0000000000010170. Epub 2020 Jul 1. Neurology. 2020. PMID: 32611643 Free PMC article.
32 results