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 | PMC:PMC10681826 | DOI:10.3349/ymj.2023.0080
Source: Yonsei Medical Journal - Category: Universities & Medical Training Authors: Source Type: research