Safety and Efficacy of Nusinersen Focusing on Renal and Hematological Parameters in Spinal Muscular Atrophy
ABSTRACT Background Spinal muscular atrophy (SMA) is a motor neuron disease caused by mutations in the SMN1 gene. Nusinersen, an antisense oligonucleotide, has been shown to improve motor function in SMA patients. However, concerns regarding its renal safety remain as previous studies have linked si...
Váldodahkkit: | , , , , , , |
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Materiálatiipa: | Artihkal |
Giella: | English |
Almmustuhtton: |
Wiley
2025-01-01
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Ráidu: | Brain and Behavior |
Fáttát: | |
Liŋkkat: | https://doi.org/10.1002/brb3.70221 |