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Mapping the differences in care for 5,000 spinal muscular atrophy patients, a survey of 24 national registries in North America, Australasia and Europe.
Bladen CL, Thompson R, Jackson JM, Garland C, Wegel C, Ambrosini A, Pisano P, Walter MC, Schreiber O, Lusakowska A, Jedrzejowska M, Kostera-Pruszczyk A, van der Pol L, Wadman RI, Gredal O, Karaduman A, Topaloglu H, Yilmaz O, Matyushenko V, Rasic VM, Kosac A, Karcagi V, Garami M, Herczegfalvi A, Monges S, Moresco A, Chertkoff L, Chamova T, Guergueltcheva V, Butoianu N, Craiu D, Korngut L, Campbell C, Haberlova J, Strenkova J, Alejandro M, Jimenez A, Ortiz GG, Enriquez GV, Rodrigues M, Roxburgh R, Dawkins H, Youngs L, Lahdetie J, Angelkova N, Saugier-Veber P, Cuisset JM, Bloetzer C, Jeannet PY, Klein A, Nascimento A, Tizzano E, Salgado D, Mercuri E, Sejersen T, Kirschner J, Rafferty K, Straub V, Bushby K, Verschuuren J, Beroud C, Lochmüller H. Bladen CL, et al. Among authors: sejersen t. J Neurol. 2014 Jan;261(1):152-63. doi: 10.1007/s00415-013-7154-1. Epub 2013 Oct 27. J Neurol. 2014. PMID: 24162038
Economic Costs of Myasthenia Gravis: A Systematic Review.
Landfeldt E, Pogoryelova O, Sejersen T, Zethraeus N, Breiner A, Lochmüller H. Landfeldt E, et al. Among authors: sejersen t. Pharmacoeconomics. 2020 Jul;38(7):715-728. doi: 10.1007/s40273-020-00912-8. Pharmacoeconomics. 2020. PMID: 32363541
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.
Kirschner J, Butoianu N, Goemans N, Haberlova J, Kostera-Pruszczyk A, Mercuri E, van der Pol WL, Quijano-Roy S, Sejersen T, Tizzano EF, Ziegler A, Servais L, Muntoni F. Kirschner J, et al. Among authors: sejersen t. Eur J Paediatr Neurol. 2020 Sep;28:38-43. doi: 10.1016/j.ejpn.2020.07.001. Epub 2020 Jul 9. Eur J Paediatr Neurol. 2020. PMID: 32763124 Free PMC article.
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.
Campbell C, Barohn RJ, Bertini E, Chabrol B, Comi GP, Darras BT, Finkel RS, Flanigan KM, Goemans N, Iannaccone ST, Jones KJ, Kirschner J, Mah JK, Mathews KD, McDonald CM, Mercuri E, Nevo Y, Péréon Y, Renfroe JB, Ryan MM, Sampson JB, Schara U, Sejersen T, Selby K, Tulinius M, Vílchez JJ, Voit T, Wei LJ, Wong BL, Elfring G, Souza M, McIntosh J, Trifillis P, Peltz SW, Muntoni F; PTC124-GD-007-DMD Study Group; ACT DMD Study Group; Clinical Evaluator Training Groups. Campbell C, et al. Among authors: sejersen t. J Comp Eff Res. 2020 Oct;9(14):973-984. doi: 10.2217/cer-2020-0095. Epub 2020 Aug 27. J Comp Eff Res. 2020. PMID: 32851872 Free article.
Costs of Illness of Spinal Muscular Atrophy: A Systematic Review.
Landfeldt E, Pechmann A, McMillan HJ, Lochmüller H, Sejersen T. Landfeldt E, et al. Among authors: sejersen t. Appl Health Econ Health Policy. 2021 Jul;19(4):501-520. doi: 10.1007/s40258-020-00624-2. Epub 2021 Feb 12. Appl Health Econ Health Policy. 2021. PMID: 33576939 Free PMC article.
Response to letter: A decision for life - Treatment decisions in newly diagnosed families with spinal muscular atrophy.
Kirschner J, Butoianu N, Goemans N, Haberlova J, Kostera-Pruszczyk A, Mercuri E, van der Pol WL, Quijano-Roy S, Sejersen T, Tizzano EF, Ziegler A, Servais L, Muntoni F. Kirschner J, et al. Among authors: sejersen t. Eur J Paediatr Neurol. 2021 Jan;30:103-104. doi: 10.1016/j.ejpn.2020.12.005. Epub 2020 Dec 14. Eur J Paediatr Neurol. 2021. PMID: 33640649 No abstract available.
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.
131 results