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

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

•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.

Bibliographic Details
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: Online
Language:English
Published: Pergamon Press 2016
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4762230/

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4762230/

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