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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...

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Detalles Bibliográficos
Autores principales: Nakao, Sayo, Yamada, Shinichi, Tsuda, Katsuya, Yokomizo, Taishi, Sato, Teruyuki, Tanoue, Shuichi, Hiraki, Teruyuki
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2020
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