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

Z. Koeks; C.L. Bladen; D. Salgado; E. van Zwet; O. Pogoryelova; G. McMacken; S. Monges; M.E. Foncuberta; K. Kekou; K. Kosma; H. Dawkins; L. Lamont; M.I. Bellgard; A.J. Roy; T. Chamova; V. Guergueltcheva; S. Chan; L. Korngut; C. Campbell; Y. Dai; J. Wang; N. Barišić; P. Brabec; J. Lähdetie; M.C. Walter; O. Schreiber-Katz; V. Karcagi; M. Garami; A. Herczegfalvi; V. Viswanathan; F. Bayat; F. Buccella; A. Ferlini; E. Kimura; J.C. van den Bergen; M. Rodrigues; R. Roxburgh; A. Lusakowska; A. Kostera-Pruszczyk; R. Santos; E. Neagu; S. Artemieva; V.M. Rasic; D. Vojinovic; M. Posada; C. Bloetzer; A. Klein; J. Díaz-Manera; E. Gallardo; A.A. Karaduman; T. Oznur; H. Topaloğlu; R. El Sherif; A. Stringer; A.V. Shatillo; A.S. Martin; H.L. Peay; J. Kirschner; K.M. Flanigan; V. Straub; K. Bushby; C. Béroud; J.J. Verschuuren; H. Lochmüller

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

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Date

2017

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Articles

Périmètre

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Langue

Anglais

Identifiant

doi: 10.3233/JND-170280

Source

Serveur académique Lausannois

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Ce document est lié à :
info:eu-repo/semantics/altIdentifier/doi/10.3233/JND-170280

Ce document est lié à :
info:eu-repo/semantics/altIdentifier/pmid/29125504

Ce document est lié à :
info:eu-repo/semantics/altIdentifier/pissn/2214-3599

Ce document est lié à :
info:eu-repo/semantics/altIdentifier/urn/urn:nbn:ch:serval-BIB_0337409C22AE5

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Serveur Académique Lausannois

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Université de Lausanne et CHUV

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info:eu-repo/semantics/openAccess , Copying allowed only for non-profit organizations , https://serval.unil.ch/disclaimer

Mots-clés 0

Adolescent; Adrenal Cortex Hormones/therapeutic use; Adult; Child; Child, Preschool; Cross-Sectional Studies; Databases as Topic; Humans; Infant; Infant, Newborn; Male; Muscular Dystrophy, Duchenne/epidemiology; Muscular Dystrophy, Duchenne/genetics; Muscular Dystrophy, Duchenne/therapy; Treatment Outcome; Young Adult; DMD; Duchenne muscular dystrophy; Neuromuscular diseases; TREAT-NMD

Sujets proches En

Erb's dystrophy Pseudohypertrophic muscular dystrophy Duchenne's muscular dystrophy Zimmerlin's dystrophy Pseudohypertrophic infantile muscular dystrophy Muscular dystrophies

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Z. Koeks et al., « Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database. », Serveur académique Lausannois, ID : 10.3233/JND-170280

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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. To determine the effects of corticosteroids on major clinical outcomes of DMD in a large multinational cohort of genetically confirmed DMD patients. In this cross-sectional study we analysed clinical data from 5345 genetically confirmed DMD patients from 31 countries held within the TREAT-NMD global DMD database. For analysis patients were categorised by corticosteroid background and further stratified by age. Loss of ambulation in non-steroid treated patients was 10 years and in corticosteroid treated patients 13 years old (p = 0.0001). Corticosteroid treated patients were less likely to need scoliosis surgery (p

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

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