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Page 1
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.
McDonald CM, Marbán E, Hendrix S, Hogan N, Ruckdeschel Smith R, Eagle M, Finkel RS, Tian C, Janas J, Harmelink MM, Varadhachary AS, Taylor MD, Hor KN, Mayer OH, Henricson EK, Furlong P, Ascheim DD, Rogy S, Williams P, Marbán L; HOPE-2 Study Group. McDonald CM, et al. Lancet. 2022 Mar 12;399(10329):1049-1058. doi: 10.1016/S0140-6736(22)00012-5. Lancet. 2022. PMID: 35279258 Free article. Clinical Trial.
DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials.
Muntoni F, Signorovitch J, Sajeev G, Lane H, Jenkins M, Dieye I, Ward SJ, McDonald C, Goemans N, Niks EH, Wong B, Servais L, Straub V, Guglieri M, de Groot IJM, Chesshyre M, Tian C, Manzur AY, Mercuri E, Aartsma-Rus A; Association Française Contre Les Myopathies; on behalf of Universitaire Ziekenhuizen Leuven Group, PRO-DMD-01, The UK NorthStar Clinical Network, CCHMC, and The DMD Italian Group. Muntoni F, et al. Neurology. 2023 Apr 11;100(15):e1540-e1554. doi: 10.1212/WNL.0000000000201626. Epub 2023 Feb 1. Neurology. 2023. PMID: 36725339 Free PMC article.
Comparison of Pulmonary Function Decline in Steroid-Treated and Steroid-Naïve Patients with Duchenne Muscular Dystrophy.
Sawnani H, Horn PS, Wong B, Darmahkasih A, Rybalsky I, Shellenbarger KC, Tian C, Rutter MM, Simakajornboon N, Amin R, Gurbani N, Pascoe J, Burrows C, Khirani S, Amaddeo A, Fauroux B. Sawnani H, et al. Among authors: rybalsky i. J Pediatr. 2019 Jul;210:194-200.e2. doi: 10.1016/j.jpeds.2019.02.037. Epub 2019 Apr 5. J Pediatr. 2019. PMID: 30955791
17 results