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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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Main Authors: | , , , , , , , , , , , , , , , , , , , , , , , |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
Lippincott Williams & Wilkins
2014
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Assuntos: | |
Acesso em linha: | 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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