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Prospective cohort study of spinal muscular atrophy types 2 and 3

OBJECTIVE: To characterize the natural history of spinal muscular atrophy type 2 and type 3 (SMA 2/3) beyond 1 year and to report data on clinical and biological outcomes for use in trial planning. METHODS: We conducted a prospective observational cohort study of 79 children and young adults with SM...

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Main Authors: Kaufmann, Petra, McDermott, Michael P., Darras, Basil T., Finkel, Richard S., Sproule, Douglas M., Kang, Peter B., Oskoui, Maryam, Constantinescu, Andrei, Gooch, Clifton L., Foley, A. Reghan, Yang, Michele L., Tawil, Rabi, Chung, Wendy K., Martens, William B., Montes, Jacqueline, Battista, Vanessa, O'Hagen, Jessica, Dunaway, Sally, Flickinger, Jean, Quigley, Janet, Riley, Susan, Glanzman, Allan M., Benton, Maryjane, Ryan, Patricia A., Punyanitya, Mark, Montgomery, Megan J., Marra, Jonathan, Koo, Benjamin, De Vivo, Darryl C.
Formato: Artigo
Idioma:Inglês
Publicado: Lippincott Williams & Wilkins 2012
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3525313/
https://ncbi.nlm.nih.gov/pubmed/23077013
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0b013e318271f7e4
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