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Intrathecal administration of nusinersen for spinal muscular atrophy: report of three cases with severe spinal deformity
BACKGROUND: Spinal muscular atrophy (SMA) is a genetic disease characterized by degeneration of the spinal cord, resulting in progressive muscle atrophy. Recently, nusinersen has been approved for treating SMA, which should be administered intrathecally. CASE PRESENTATION: Patient 1 was a 36-year-ol...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Berlin Heidelberg
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7171013/ https://www.ncbi.nlm.nih.gov/pubmed/32314319 http://dx.doi.org/10.1186/s40981-020-00334-7 |