Observational Study of Spinal Muscular Atrophy Type 2 and 3: Functional Outcomes Over 1 Year

OBJECTIVE: To characterize the short-term course of spinal muscular atrophy (SMA) in a genetically and clinically well-defined cohort of patients with SMA. DESIGN: A comprehensive multicenter, longitudinal, observational study. SETTING: The Pediatric Neuromuscular Clinical Research Network for SMA,...

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Main Authors: Kaufmann, Petra, McDermott, Michael P., Darras, Basil T., Finkel, Richard, Kang, Peter, Oskoui, Maryam, Constantinescu, Andrei, Sproule, Douglas Michael, Foley, A. Reghan, Yang, Michele, Tawil, Rabi, Chung, Wendy, Martens, Bill, Montes, Jacqueline, O'Hagen, Jessica, Dunaway, Sally, Flickinger, Jean M., Quigley, Janet, Riley, Susan, Glanzman, Allan M., Benton, Maryjane, Ryan, Patricia A., Irvine, Carrie, Annis, Christine L., Butler, Hailly, Caracciolo, Jayson, Montgomery, Megan, Marra, Jonathan, Koo, Benjamin, De Vivo, Darryl C.
Format: Artigo
Language:Inglês
Published: 2011
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3839315/
https://ncbi.nlm.nih.gov/pubmed/21320981
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/archneurol.2010.373
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