Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy

Highlights • This paper shows that DMD boys could be grouped into classes sharing trajectories. • Using analysis accounting for trajectory classes, the variation was strongly reduced. • Reducing unexplained variation could help to improve DMD clinical trial design.

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Bibliographische Detailangaben
Veröffentlicht in:	Neuromuscular disorders : NMD 2016-09, Vol.26 (9), p.576-583
Hauptverfasser:	Mercuri, Eugenio, Signorovitch, James Edward, Swallow, Elyse, Song, Jinlin, Ward, Susan J
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Age Factors Ambulatory function Child Child, Preschool Disease Progression Duchenne muscular dystrophy Exercise Test Follow-Up Studies Humans Latent class trajectory analysis Male Muscular Dystrophy, Duchenne - classification Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - physiopathology Natural history Neurology Retrospective Studies Steroids - therapeutic use Variability Walking
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Beschreibung
Zusammenfassung:	Highlights • This paper shows that DMD boys could be grouped into classes sharing trajectories. • Using analysis accounting for trajectory classes, the variation was strongly reduced. • Reducing unexplained variation could help to improve DMD clinical trial design.
ISSN:	0960-8966 1873-2364
DOI:	10.1016/j.nmd.2016.05.016