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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Bibliografiske detaljer
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.
Format: Artigo
Sprog:Inglês
Udgivet: Lippincott Williams & Wilkins 2012
Fag:
Articles
Online adgang: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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