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121 results

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Page 1
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG).
Finkel RS, Darras BT, Mendell JR, Day JW, Kuntz NL, Connolly AM, Zaidman CM, Crawford TO, Butterfield RJ, Shieh PB, Tennekoon G, Brandsema JF, Iannaccone ST, Shoffner J, Kavanagh S, Macek TA, Tauscher-Wisniewski S. Finkel RS, et al. Among authors: tennekoon g. J Neuromuscul Dis. 2023;10(3):389-404. doi: 10.3233/JND-221560. J Neuromuscul Dis. 2023. PMID: 36911944 Free PMC article.
Step Activity Monitoring in Boys with Duchenne Muscular Dystrophy and its Correlation with Magnetic Resonance Measures and Functional Performance.
Nair KS, Lott DJ, Forbes SC, Barnard AM, Willcocks RJ, Senesac CR, Daniels MJ, Harrington AT, Tennekoon GI, Zilke K, Finanger EL, Finkel RS, Rooney WD, Walter GA, Vandenborne K. Nair KS, et al. Among authors: tennekoon gi. J Neuromuscul Dis. 2022;9(3):423-436. doi: 10.3233/JND-210746. J Neuromuscul Dis. 2022. PMID: 35466946 Free PMC article.
Development of Contractures in DMD in Relation to MRI-Determined Muscle Quality and Ambulatory Function.
Willcocks RJ, Barnard AM, Wortman RJ, Senesac CR, Lott DJ, Harrington AT, Zilke KL, Forbes SC, Rooney WD, Wang DJ, Finanger EL, Tennekoon GI, Daniels MJ, Triplett WT, Walter GA, Vandenborne K. Willcocks RJ, et al. Among authors: tennekoon gi. J Neuromuscul Dis. 2022;9(2):289-302. doi: 10.3233/JND-210731. J Neuromuscul Dis. 2022. PMID: 35124659 Free PMC article.
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.
MLIP causes recessive myopathy with rhabdomyolysis, myalgia and baseline elevated  serum creatine kinase.
Lopes Abath Neto O, Medne L, Donkervoort S, Rodríguez-García ME, Bolduc V, Hu Y, Guadagnin E, Foley AR, Brandsema JF, Glanzman AM, Tennekoon GI, Santi M, Berger JH, Megeney LA, Komaki H, Inoue M, Cotrina-Vinagre FJ, Hernández-Lain A, Martin-Hernández E, Williams L, Borell S, Schorling D, Lin K, Kolokotronis K, Lichter-Konecki U, Kirschner J, Nishino I, Banwell B, Martínez-Azorín F, Burgon PG, Bönnemann CG. Lopes Abath Neto O, et al. Among authors: tennekoon gi. Brain. 2021 Oct 22;144(9):2722-2731. doi: 10.1093/brain/awab275. Brain. 2021. PMID: 34581780 Free PMC article.
Psychometric properties of the PEDI-CAT for children and youth with spinal muscular atrophy.
Fragala-Pinkham M, Pasternak A, McDermott MP, Mirek E, Glanzman AM, Montes J, Dunaway Young S, Salazar R, Quigley J, Riley SO, Chiriboga CA, Finkel RS, Tennekoon G, Martens WB, De Vivo DC, Darras BT. Fragala-Pinkham M, et al. Among authors: tennekoon g. J Pediatr Rehabil Med. 2021;14(3):451-461. doi: 10.3233/PRM-190664. J Pediatr Rehabil Med. 2021. PMID: 34275913
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: tennekoon g. 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.
Walking activity in a large cohort of boys with Duchenne muscular dystrophy.
Lott DJ, Taivassalo T, Senesac CR, Willcocks RJ, Harrington AM, Zilke K, Cunkle H, Powers C, Finanger EL, Rooney WD, Tennekoon GI, Vandenborne K. Lott DJ, et al. Among authors: tennekoon gi. Muscle Nerve. 2021 Feb;63(2):192-198. doi: 10.1002/mus.27119. Epub 2020 Nov 27. Muscle Nerve. 2021. PMID: 33188573 Free PMC article.
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.
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: tennekoon gi. Radiology. 2020 Jun;295(3):616-625. doi: 10.1148/radiol.2020192210. Epub 2020 Apr 14. Radiology. 2020. PMID: 32286193 Free PMC article.
121 results