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Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0.
Cicala G, Pane M, Coratti G, Brogna C, Fanelli L, Norcia G, Forcina N, Mazzone E, Stanca G, Ferrante R, Vento A, Ferraroli E, Ricci M, Capasso A, Leone D, Palermo C, Berti B, Cutrona C, Mahyew A, Duong T, Goemans N, Vroom E, Mercuri E. Cicala G, et al. Among authors: vroom e. Neuromuscul Disord. 2023 Sep;33(9):69-73. doi: 10.1016/j.nmd.2023.07.003. Epub 2023 Jul 22. Neuromuscul Disord. 2023. PMID: 37612177 Free article.
Towards harmonisation of outcome measures for DMD and SMA within TREAT-NMD; report of three expert workshops: TREAT-NMD/ENMC workshop on outcome measures, 12th--13th May 2007, Naarden, The Netherlands; TREAT-NMD workshop on outcome measures in experimental trials for DMD, 30th June--1st July 2007, Naarden, The Netherlands; conjoint Institute of Myology TREAT-NMD meeting on physical activity monitoring in neuromuscular disorders, 11th July 2007, Paris, France.
Mercuri E, Mayhew A, Muntoni F, Messina S, Straub V, Van Ommen GJ, Voit T, Bertini E, Bushby K; TREAT-NMD Neuromuscular Network. Mercuri E, et al. Neuromuscul Disord. 2008 Nov;18(11):894-903. doi: 10.1016/j.nmd.2008.07.003. Epub 2008 Sep 24. Neuromuscul Disord. 2008. PMID: 18818076 No abstract available.
Transition in Duchenne muscular dystrophy: An expert meeting report and description of transition needs in an emergent patient population: (Parent Project Muscular Dystrophy Transition Expert Meeting 17-18 June 2011, Amsterdam, The Netherlands).
Schrans DG, Abbott D, Peay HL, Pangalila RF, Vroom E, Goemans N, Vles JS, Aldenkamp AP, Hendriksen JG; Expert Meeting Participants on Transition and Adulthood in Duchenne Muscular Dystrophy. Schrans DG, et al. Among authors: vroom e. Neuromuscul Disord. 2013 Mar;23(3):283-6. doi: 10.1016/j.nmd.2012.08.009. Epub 2012 Sep 16. Neuromuscul Disord. 2013. PMID: 22989602 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: vroom e. 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.
Biomarkers and surrogate endpoints in Duchenne: meeting report.
Aartsma-Rus A, Ferlini A, Vroom E. Aartsma-Rus A, et al. Among authors: vroom e. Neuromuscul Disord. 2014 Aug;24(8):743-5. doi: 10.1016/j.nmd.2014.03.006. Epub 2014 May 28. Neuromuscul Disord. 2014. PMID: 24951452 No abstract available.
Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy.
Lynn S, Aartsma-Rus A, Bushby K, Furlong P, Goemans N, De Luca A, Mayhew A, McDonald C, Mercuri E, Muntoni F, Pohlschmidt M, Verschuuren J, Voit T, Vroom E, Wells DJ, Straub V. Lynn S, et al. Among authors: vroom e. Neuromuscul Disord. 2015 Jan;25(1):96-105. doi: 10.1016/j.nmd.2014.09.003. Epub 2014 Sep 11. Neuromuscul Disord. 2015. PMID: 25307856 No abstract available.
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.
Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A, Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A. Straub V, et al. Among authors: vroom e. Lancet Neurol. 2016 Jul;15(8):882-890. doi: 10.1016/S1474-4422(16)30035-7. Lancet Neurol. 2016. PMID: 27302365 Review.
83 results