Safety and Efficacy of Nusinersen Focusing on Renal and Hematological Parameters in Spinal Muscular Atrophy

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...

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Main Authors: Hüseyin Bahadır Şenol, Gizem Yıldız, Ayşe İpek Polat, Adem Aydın, Ayşe Semra Hız, Alper Soylu, Uluç Yiş
Format: Article
Language:English
Published: Wiley 2025-01-01
Series:Brain and Behavior
Subjects:
antisense oligonucleotide
spinal muscular atrophy
urine creatinine
urine protein
Online Access:https://doi.org/10.1002/brb3.70221
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