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Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy.
Servais L, Straathof CSM, Schara U, Klein A, Leinonen M, Hasham S, Meier T, De Waele L, Gordish-Dressman H, McDonald CM, Mayer OH, Voit T, Mercuri E, Buyse GM; SYROS and CINRG DNHS Investigators. Servais L, et al. Among authors: klein a. Neuromuscul Disord. 2020 Jan;30(1):5-16. doi: 10.1016/j.nmd.2019.10.008. Epub 2019 Nov 5. Neuromuscul Disord. 2020. PMID: 31813614 Free article. Clinical Trial.
Novel mutations expand the clinical spectrum of DYNC1H1-associated spinal muscular atrophy.
Scoto M, Rossor AM, Harms MB, Cirak S, Calissano M, Robb S, Manzur AY, Martínez Arroyo A, Rodriguez Sanz A, Mansour S, Fallon P, Hadjikoumi I, Klein A, Yang M, De Visser M, Overweg-Plandsoen WC, Baas F, Taylor JP, Benatar M, Connolly AM, Al-Lozi MT, Nixon J, de Goede CG, Foley AR, Mcwilliam C, Pitt M, Sewry C, Phadke R, Hafezparast M, Chong WK, Mercuri E, Baloh RH, Reilly MM, Muntoni F. Scoto M, et al. Among authors: klein a. Neurology. 2015 Feb 17;84(7):668-79. doi: 10.1212/WNL.0000000000001269. Epub 2015 Jan 21. Neurology. 2015. PMID: 25609763 Free PMC article.
Quantitative muscle MRI: A powerful surrogate outcome measure in Duchenne muscular dystrophy.
Bonati U, Hafner P, Schädelin S, Schmid M, Naduvilekoot Devasia A, Schroeder J, Zuesli S, Pohlman U, Neuhaus C, Klein A, Sinnreich M, Haas T, Gloor M, Bieri O, Fischmann A, Fischer D. Bonati U, et al. Among authors: klein a. Neuromuscul Disord. 2015 Sep;25(9):679-85. doi: 10.1016/j.nmd.2015.05.006. Epub 2015 Jun 4. Neuromuscul Disord. 2015. PMID: 26096788
Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype.
Servais L, Montus M, Guiner CL, Ben Yaou R, Annoussamy M, Moraux A, Hogrel JY, Seferian AM, Zehrouni K, Le Moing AG, Gidaro T, Vanhulle C, Laugel V, Butoianu N, Cuisset JM, Sabouraud P, Cances C, Klein A, Leturcq F, Moullier P, Voit T. Servais L, et al. Among authors: klein a. J Neuromuscul Dis. 2015 Sep 2;2(3):269-279. doi: 10.3233/JND-150100. J Neuromuscul Dis. 2015. PMID: 27858743 Free PMC article.
Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database.
Koeks Z, Bladen CL, Salgado D, van Zwet E, Pogoryelova O, McMacken G, Monges S, Foncuberta ME, Kekou K, Kosma K, Dawkins H, Lamont L, Bellgard MI, Roy AJ, Chamova T, Guergueltcheva V, Chan S, Korngut L, Campbell C, Dai Y, Wang J, Barišić N, Brabec P, Lähdetie J, Walter MC, Schreiber-Katz O, Karcagi V, Garami M, Herczegfalvi A, Viswanathan V, Bayat F, Buccella F, Ferlini A, Kimura E, van den Bergen JC, Rodrigues M, Roxburgh R, Lusakowska A, Kostera-Pruszczyk A, Santos R, Neagu E, Artemieva S, Rasic VM, Vojinovic D, Posada M, Bloetzer C, Klein A, Díaz-Manera J, Gallardo E, Karaduman AA, Oznur T, Topaloğlu H, El Sherif R, Stringer A, Shatillo AV, Martin AS, Peay HL, Kirschner J, Flanigan KM, Straub V, Bushby K, Béroud C, Verschuuren JJ, Lochmüller H. Koeks Z, et al. Among authors: klein a. J Neuromuscul Dis. 2017;4(4):293-306. doi: 10.3233/JND-170280. J Neuromuscul Dis. 2017. PMID: 29125504 Free PMC article.
Timed function tests, motor function measure, and quantitative thigh muscle MRI in ambulant children with Duchenne muscular dystrophy: A cross-sectional analysis.
Schmidt S, Hafner P, Klein A, Rubino-Nacht D, Gocheva V, Schroeder J, Naduvilekoot Devasia A, Zuesli S, Bernert G, Laugel V, Bloetzer C, Steinlin M, Capone A, Gloor M, Tobler P, Haas T, Bieri O, Zumbrunn T, Fischer D, Bonati U. Schmidt S, et al. Among authors: klein a. Neuromuscul Disord. 2018 Jan;28(1):16-23. doi: 10.1016/j.nmd.2017.10.003. Epub 2017 Nov 21. Neuromuscul Disord. 2018. PMID: 29174526 Clinical Trial.
4,640 results