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)

1 GMGF - Génétique Médicale et Génomique Fonctionnelle
2 Department of Medical Statistics and Bioinformatics
3 Neuropaediatrics
4 Centre for Comparative Genomics
5 Department of Biological and Environmental Engineering
6 Department of Reproduction and Growth, UOL of Medical Genetics (University Hospital St Anna, Ferrara)
7 Department of Embryology [Warsaw]
8 Medicina Pediátrica y del Desarrollo
9 Department of Neurology
10 Newcastle University [Newcastle]

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.

Keywords: Neuromuscular diseases; Duchenne muscular dystrophy; DMD; TREAT-NMD

Domains: Bioinformatics [q-bio.QM]; Human genetics; Molecular biology

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, 2017, 4 (4), pp.293 - 306. ⟨10.3233/JND-170280⟩. ⟨hal-01681734⟩