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Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.
Shieh PB, Elfring G, Trifillis P, Santos C, Peltz SW, Parsons JA, Apkon S, Darras BT, Campbell C, McDonald CM; Members of the Ataluren Phase IIb Study Group; Members of the Ataluren Phase IIb Study Clinical Evaluator Training Group; Members of the ACT DMD Study Group; Members of the ACT DMD Clinical Evaluator Training Group. Shieh PB, et al. J Comp Eff Res. 2021 Dec;10(18):1337-1347. doi: 10.2217/cer-2021-0018. Epub 2021 Oct 25. J Comp Eff Res. 2021. PMID: 34693725 Free article.
Disease-modifying effects of edasalonexent, an NF-κB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trial.
Finkel RS, Finanger E, Vandenborne K, Sweeney HL, Tennekoon G, Shieh PB, Willcocks R, Walter G, Rooney WD, Forbes SC, Triplett WT, Yum SW, Mancini M, MacDougall J, Fretzen A, Bista P, Nichols A, Donovan JM. Finkel RS, et al. Among authors: sweeney hl. Neuromuscul Disord. 2021 May;31(5):385-396. doi: 10.1016/j.nmd.2021.02.001. Epub 2021 Feb 4. Neuromuscul Disord. 2021. PMID: 33678513 Free 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. Neurology. 2020 Sep 8;95(10):e1381-e1391. doi: 10.1212/WNL.0000000000010170. Epub 2020 Jul 1. Neurology. 2020. PMID: 32611643 Free PMC article.
Myosin Structures.
Sweeney HL, Houdusse A, Robert-Paganin J. Sweeney HL, et al. Adv Exp Med Biol. 2020;1239:7-19. doi: 10.1007/978-3-030-38062-5_2. Adv Exp Med Biol. 2020. PMID: 32451853 Review.
Upper and Lower Extremities in Duchenne Muscular Dystrophy Evaluated with Quantitative MRI and Proton MR Spectroscopy in a Multicenter Cohort.
Forbes SC, Arora H, Willcocks RJ, Triplett WT, Rooney WD, Barnard AM, Alabasi U, Wang DJ, Lott DJ, Senesac CR, Harrington AT, Finanger EL, Tennekoon GI, Brandsema J, Daniels MJ, Sweeney HL, Walter GA, Vandenborne K. Forbes SC, et al. Among authors: sweeney hl. Radiology. 2020 Jun;295(3):616-625. doi: 10.1148/radiol.2020192210. Epub 2020 Apr 14. Radiology. 2020. PMID: 32286193 Free PMC article.
262 results