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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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: 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.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Lippincott Williams & Wilkins 2014
Pynciau:
Article
Mynediad Ar-lein: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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