Observational study of spinal muscular atrophy type I and implications for clinical trials

OBJECTIVES: Prospective cohort study to characterize the clinical features and course of spinal muscular atrophy type I (SMA-I). METHODS: Patients were enrolled at 3 study sites and followed for up to 36 months with serial clinical, motor function, laboratory, and electrophysiologic outcome assessme...

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Päätekijät: Finkel, Richard S., McDermott, Michael P., Kaufmann, Petra, Darras, Basil T., Chung, Wendy K., Sproule, Douglas M., Kang, Peter B., Foley, A. Reghan, Yang, Michelle L., Martens, William B., Oskoui, Maryam, Glanzman, Allan M., Flickinger, Jean, Montes, Jacqueline, Dunaway, Sally, O'Hagen, Jessica, Quigley, Janet, Riley, Susan, Benton, Maryjane, Ryan, Patricia A., Montgomery, Megan, Marra, Jonathan, Gooch, Clifton, De Vivo, Darryl C.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Lippincott Williams & Wilkins 2014
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Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC4155049/
https://ncbi.nlm.nih.gov/pubmed/25080519
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000000741
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