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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Detaylı Bibliyografya
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Lippincott Williams & Wilkins 2014
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Article
Online Erişim: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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