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255 results

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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: servais l. 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.
International workshop on assessment of upper limb function in Duchenne Muscular Dystrophy: Rome, 15-16 February 2012.
Mercuri E, McDonald C, Mayhew A, Florence J, Mazzone E, Bianco F, Decostre V, Servais L, Ricotti V, Goemans N, Vroom E. Mercuri E, et al. Among authors: servais l. Neuromuscul Disord. 2012 Nov;22(11):1025-8. doi: 10.1016/j.nmd.2012.06.006. Epub 2012 Jul 13. Neuromuscul Disord. 2012. PMID: 22795657 Free PMC article. No abstract available.
Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy.
Mayhew A, Mazzone ES, Eagle M, Duong T, Ash M, Decostre V, Vandenhauwe M, Klingels K, Florence J, Main M, Bianco F, Henrikson E, Servais L, Campion G, Vroom E, Ricotti V, Goemans N, McDonald C, Mercuri E; Performance of the Upper Limb Working Group. Mayhew A, et al. Among authors: servais l. Dev Med Child Neurol. 2013 Nov;55(11):1038-45. doi: 10.1111/dmcn.12213. Epub 2013 Aug 1. Dev Med Child Neurol. 2013. PMID: 23902233 Free article. Review.
Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial.
Flanigan KM, Voit T, Rosales XQ, Servais L, Kraus JE, Wardell C, Morgan A, Dorricott S, Nakielny J, Quarcoo N, Liefaard L, Drury T, Campion G, Wright P. Flanigan KM, et al. Among authors: servais l. Neuromuscul Disord. 2014 Jan;24(1):16-24. doi: 10.1016/j.nmd.2013.09.004. Epub 2013 Sep 11. Neuromuscul Disord. 2014. PMID: 24321374 Free PMC article. Clinical Trial.
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.
Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, Campion G, De Kimpe SJ, Eagle M, Guglieri M, Hood S, Liefaard L, Lourbakos A, Morgan A, Nakielny J, Quarcoo N, Ricotti V, Rolfe K, Servais L, Wardell C, Wilson R, Wright P, Kraus JE. Voit T, et al. Among authors: servais l. Lancet Neurol. 2014 Oct;13(10):987-96. doi: 10.1016/S1474-4422(14)70195-4. Epub 2014 Sep 7. Lancet Neurol. 2014. PMID: 25209738 Clinical Trial.
Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies.
Rouillon J, Poupiot J, Zocevic A, Amor F, Léger T, Garcia C, Camadro JM, Wong B, Pinilla R, Cosette J, Coenen-Stass AM, Mcclorey G, Roberts TC, Wood MJ, Servais L, Udd B, Voit T, Richard I, Svinartchouk F. Rouillon J, et al. Among authors: servais l. Hum Mol Genet. 2015 Sep 1;24(17):4916-32. doi: 10.1093/hmg/ddv214. Epub 2015 Jun 9. Hum Mol Genet. 2015. PMID: 26060189 Free PMC article.
DMD and West syndrome.
Cardas R, Iliescu C, Butoianu N, Seferian A, Gataullina S, Gargaun E, Nectoux J, Bienvenu T, Craiu D, Gidaro T, Servais L. Cardas R, et al. Among authors: servais l. Neuromuscul Disord. 2017 Oct;27(10):911-913. doi: 10.1016/j.nmd.2017.07.008. Epub 2017 Jul 19. Neuromuscul Disord. 2017. PMID: 28802771
A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy.
Victor RG, Sweeney HL, Finkel R, McDonald CM, Byrne B, Eagle M, Goemans N, Vandenborne K, Dubrovsky AL, Topaloglu H, Miceli MC, Furlong P, Landry J, Elashoff R, Cox D; Tadalafil DMD Study Group. Victor RG, et al. Neurology. 2017 Oct 24;89(17):1811-1820. doi: 10.1212/WNL.0000000000004570. Epub 2017 Sep 29. Neurology. 2017. PMID: 28972192 Free PMC article. Clinical Trial.
255 results