Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials

Highlights • The paper reports for the first time patterns of progression in type 2 and 3 SMA. • Different trajectories can be identified in ambulant and non-ambulant patients. • Age appears to be an important factor in determining trajectories of progression.

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Veröffentlicht in:	Neuromuscular disorders : NMD 2016-02, Vol.26 (2), p.126-131
Hauptverfasser:	Mercuri, Eugenio, Finkel, Richard, Montes, Jacqueline, Mazzone, Elena S, Sormani, Maria Pia, Main, Marion, Ramsey, Danielle, Mayhew, Anna, Glanzman, Allan M, Dunaway, Sally, Salazar, Rachel, Pasternak, Amy, Quigley, Janet, Pane, Marika, Pera, Maria Carmela, Scoto, Mariacristina, Messina, Sonia, Sframeli, Maria, Vita, Gian Luca, D'Amico, Adele, van den Hauwe, Marleen, Sivo, Serena, Goemans, Nathalie, Kaufmann, Petra, Darras, Basil T, Bertini, Enrico, Muntoni, Francesco, De Vivo, Darryl C
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Child Child, Preschool Clinical Trials as Topic Disease Progression Female Hammersmith Functional Motor Scale Humans Male Middle Aged Mobility Limitation Neurology Outcome measures Retrospective Studies Spinal Muscular Atrophies of Childhood - physiopathology Spinal muscular atrophy Young Adult
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Beschreibung
Zusammenfassung:	Highlights • The paper reports for the first time patterns of progression in type 2 and 3 SMA. • Different trajectories can be identified in ambulant and non-ambulant patients. • Age appears to be an important factor in determining trajectories of progression.
ISSN:	0960-8966 1873-2364
DOI:	10.1016/j.nmd.2015.10.006