Nusinersen for Spinal Muscular Atrophy Type I with Chronic Respiratory Failure: A Retrospective Study in South Korea

CONCLUSION: Nusinersen is safe and effective in patients with SMA type I, even those with chronic respiratory failure and those on permanent ventilation. No significant adverse effects of nusinersen were observed.PMID:37992742 | DOI:10.3349/ymj.2023.0080

Source: Yonsei Medical Journal - November 22, 2023 Category: Universities & Medical Training Authors: Hui Jin Shin Ji-Hoon Na Hyunjoo Lee Young-Mock Lee Source Type: research