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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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Detalhes bibliográficos
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 em: Lippincott Williams & Wilkins 2012
Assuntos:
Acesso em linha: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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