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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Bibliografiske detaljer
Udgivet i:J Cell Mol Med
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2020
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Original Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7077557/
https://ncbi.nlm.nih.gov/pubmed/32032473
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/jcmm.14939
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