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Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database

Zaïda Koeks Catherine L Bladen David Salgado 1 Erik van Zwet 2 Oksana Pogoryelova Grace Mcmacken Soledad Monges 3 Maria E Foncuberta Kyriaki Kekou Konstantina Kosma Hugh Dawkins 4 Leanne Lamont Matthew I Bellgard Anna Roy Teodora Chamova Velina Guergueltcheva Sophelia Chan Lawrence Korngut Craig Campbell Yi Dai Jen Wang Nina Barišić Petr Brabec Jaana Lähdetie Maggie C Walter 5 Olivia Schreiber-Katz Veronika Karcagi Marta Garami Agnes Herczegfalvi Venkatarman Viswanathan Farhad Bayat Filippo Buccella Alessandra Ferlini 6 En Kimura Jan van den Bergen Miriam Rodrigues Richard Roxburgh Anna Lusakowska 7 Anna Kostera-Pruszczyk Rosário Santos Elena Neagu Svetlana Artemieva Vedrana Milic Rasic Dina Vojinovic Manuel Posada 8 Clemens Bloetzer Andrea Klein Jordi Díaz-Manera Eduard Gallardo a Ayşe Karaduman Tunca Oznur Haluk Topaloğlu Rasha El Sherif Angela Stringer Andriy V Shatillo Ann S Martin Holly L Peay Jan J Kirschner Kevin M Flanigan Volker Straub 9 Kate Bushby 9 Christophe Béroud 1 Jan J Verschuuren Hanns Lochmuller 10
Abstract : Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) have indicated greater disease variability in terms of progression than expected. In addition, as average life-expectancy increases, reliable data is required on clinical progression in the older DMD population.
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Zaïda Koeks, Catherine L Bladen, David Salgado, Erik van Zwet, Oksana Pogoryelova, et al.. Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database. Journal of Neuromuscular Diseases, IOS Press, 2017, 4 (4), pp.293 - 306. ⟨10.3233/JND-170280⟩. ⟨hal-01681734⟩

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