Safety analysis of laboratory parameters in paediatric patients with spinal muscular atrophy treated with nusinersen

• Main Authors: Xiaomei Zhu (Author), Hui Li (Author), Chaoping Hu (Author), Min Wu (Author), Shuizhen Zhou (Author), Yi Wang (Author), Wenhui Li (Author)
• Format: Book
• Published: BMC, 2024-07-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder that can be treated with intrathecal nusinersen, an antisense oligonucleotide. In addition to efficacy, safety is a determining factor in the success of any therapy. Here, we aim to assess the safety of nus...