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Fourteen differentially expressed miRNAs were notably modified in CSF from baseline to follow-up during treatment with nusinersen. The best magnitude of modification ended up being mentioned in miR-7-5p, miR-15a-5p, miR-15b-3p/5p, miR-126-5p, miR-128-2-5p and miR-130a-3p which encompassed a spectrum of functions predominantly in neurogenesis, neuronal differentiation and growth. The prominent signaling pathways identified in this study were the mammalian target of rapamy