Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database
- Koeks, Zaïda
- Bladen, Catherine L
- Salgado, David
- van Zwet, Erik
- Pogoryelova, Oksana
- Mcmacken, Grace
- Monges, Soledad
- Foncuberta, Maria E
- Kekou, Kyriaki
- Kosma, Konstantina
- Dawkins, Hugh
- Lamont, Leanne
- Bellgard, Matthew I
- Roy, Anna
- Chamova, Teodora
- Guergueltcheva, Velina
- Chan, Sophelia
- Korngut, Lawrence
- Campbell, Craig
- Dai, Yi
- Wang, Jen
- Barišić, Nina
- Brabec, Petr
- Lähdetie, Jaana
- Walter, Maggie C
- Schreiber-Katz, Olivia
- Karcagi, Veronika
- Garami, Marta
- Herczegfalvi, Agnes
- Viswanathan, Venkatarman
- Bayat, Farhad
- Buccella, Filippo
- Ferlini, Alessandra
- Kimura, En
- van den Bergen, Jan
- Rodrigues, Miriam
- Roxburgh, Richard
- Lusakowska, Anna
- Kostera-Pruszczyk, Anna
- Santos, Rosário
- Neagu, Elena
- Artemieva, Svetlana
- Rasic, Vedrana Milic
- Vojinovic, Dina
- Posada, Manuel
- Bloetzer, Clemens
- Klein, Andrea
- Díaz-Manera, Jordi
- Gallardo, Eduard
- Karaduman, a Ayşe
- Oznur, Tunca
- Topaloğlu, Haluk
- El Sherif, Rasha
- Stringer, Angela
- Shatillo, Andriy V
- Martin, Ann S
- Peay, Holly L
- Kirschner, Jan J
- Flanigan, Kevin M
- Straub, Volker
- Bushby, Kate
- Béroud, Christophe
- Verschuuren, Jan J
- Lochmuller, Hanns
DOIAbstract
International audienceRecent 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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