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,Erik van Zwet,Oksana Pogoryelova,Grace McMacken,Soledad Monges,Maria E Foncuberta,Kyriaki Kekou,Konstantina Kosma,Hugh Dawkins, Leanne Lamont,Matthew I Bellgard,Anna J Roy,Teodora Chamova,Velina Guergueltcheva,Sophelia Chan,Lawrence Korngut,Craig Campbell,Yi Dai,Jen Wang,Nina Barišić,Petr Brabec,Jaana Lähdetie,Maggie C Walter,Olivia Schreiber-Katz,Veronika Karcagi, Marta Garami,Agnes Herczegfalvi, Venkatarman Viswanathan,Farhad Bayat,Filippo Buccella,Alessandra Ferlini,En Kimura,Janneke C van den Bergen,Miriam Rodrigues,Richard Roxburgh,Anna Lusakowska,Anna Kostera-Pruszczyk,Rosário Santos,Elena Neagu,Svetlana Artemieva,Vedrana Milic Rasic,Dina Vojinovic,Manuel Posada,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 Kirschner,Kevin M Flanigan,Volker Straub,Kate Bushby,Christophe Béroud,Jan J Verschuuren,Hanns Lochmüller

Journal of neuromuscular diseases（2017）

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摘要

This study provides data on clinical outcomes of DMD across many healthcare settings and including a sizeable cohort of older patients. Our data confirm the benefits of corticosteroid treatment on ambulation, need for scoliosis surgery, ventilation and, to a lesser extent, cardiomyopathy. This study underlines the importance of data collection via patient registries and the critical role of multi-centre collaboration in the rare disease field.

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关键词

DMD,Duchenne muscular dystrophy,Neuromuscular diseases,TREAT-NMD

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