Nusinersen treatment and cerebrospinal fluid neurofilaments: An explorative study on Spinal Muscular Atrophy type 3 patients

The antisense oligonucleotide Nusinersen has been recently licensed to treat spinal muscular atrophy (SMA). Since SMA type 3 is characterized by variable phenotype and milder progression, biomarkers of early treatment response are urgently needed. We investigated the cerebrospinal fluid (CSF) concen...

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Detalles Bibliográficos
Autores principales: Faravelli, Irene, Meneri, Megi, Saccomanno, Domenica, Velardo, Daniele, Abati, Elena, Gagliardi, Delia, Parente, Valeria, Petrozzi, Lucia, Ronchi, Dario, Stocchetti, Nino, Calderini, Edoardo, D’Angelo, Grazia, Chidini, Giovanna, Prandi, Edi, Ricci, Giulia, Siciliano, Gabriele, Bresolin, Nereo, Comi, Giacomo Pietro, Corti, Stefania, Magri, Francesca, Govoni, Alessandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7077557/
https://www.ncbi.nlm.nih.gov/pubmed/32032473
http://dx.doi.org/10.1111/jcmm.14939

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