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Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy.
Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Contesse MG, et al. Among authors: nelson l. PLoS One. 2022 Apr 13;17(4):e0266845. doi: 10.1371/journal.pone.0266845. eCollection 2022. PLoS One. 2022. PMID: 35417501 Free PMC article.
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E; Clinical Evaluator Training Group; ACT DMD Study Group. McDonald CM, et al. Lancet. 2017 Sep 23;390(10101):1489-1498. doi: 10.1016/S0140-6736(17)31611-2. Epub 2017 Jul 17. Lancet. 2017. PMID: 28728956 Clinical Trial.
Motor Function Test Reliability During the NeuroNEXT Spinal Muscular Atrophy Infant Biomarker Study.
Krosschell KJ, Bosch M, Nelson L, Duong T, Lowes LP, Alfano LN, Benjamin D, Carry TB, Devine G, Kelley C, Gadekan R, Malkus EC, Pasternak A, Provance-Orr S, Roemeiser-Logan L, Nicorici A, Trussell D, Young SD, Fetterman JR, Montes J, Powers PJ, Quinones R, Quigley J, Coffey CS, Yankey JW, Bartlett A, Kissel JT, Kolb SJ; NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators. Krosschell KJ, et al. Among authors: nelson l. J Neuromuscul Dis. 2018;5(4):509-521. doi: 10.3233/JND-180327. J Neuromuscul Dis. 2018. PMID: 30223401 Free PMC article.
Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.
Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR; MDA DMD Clinical Research Network. Connolly AM, et al. Among authors: nelson ll. Muscle Nerve. 2019 Jun;59(6):650-657. doi: 10.1002/mus.26441. Epub 2019 Feb 23. Muscle Nerve. 2019. PMID: 30706490
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.
SMA-MAP: a plasma protein panel for spinal muscular atrophy.
Kobayashi DT, Shi J, Stephen L, Ballard KL, Dewey R, Mapes J, Chung B, McCarthy K, Swoboda KJ, Crawford TO, Li R, Plasterer T, Joyce C; Biomarkers for Spinal Muscular Atrophy Study Group; Chung WK, Kaufmann P, Darras BT, Finkel RS, Sproule DM, Martens WB, McDermott MP, De Vivo DC; Pediatric Neuromuscular Clinical Research Network; Walker MG, Chen KS. Kobayashi DT, et al. PLoS One. 2013;8(4):e60113. doi: 10.1371/journal.pone.0060113. Epub 2013 Apr 2. PLoS One. 2013. PMID: 23565191 Free PMC article.
3,159 results