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

The aim of the study was to establish 12-month changes in the Hammersmith Functional motor scale in a large cohort of SMA patients, to identify patterns of disease progression and the effect of different variables. 268 patients were included in this multicentric study. Their age ranged between 2.5 a...

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Published in:Neuromuscul Disord
Main Authors: 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: Artigo
Language:Inglês
Published: Pergamon Press 2016
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4762230/
https://ncbi.nlm.nih.gov/pubmed/26776503
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nmd.2015.10.006
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