Real-world analysis of the efficacy and safety of nusinersen in pediatric patients with spinal muscular atrophy
Nusinersen demonstrated significant clinical efficacy and favorable safety in pediatric SMA patients, with improved economic feasibility after insurance coverage. Our findings support early SMA screening and presymptomatic nusinersen administration to maximize therapeutic benefits. Further multicenter, large-sample, long-term follow-up studies are warranted to validate and expand upon these findings.
Authors
- Jin Xu (ORCID: https://orcid.org/0000-0003-0586-6084)
- Wenjing Li (ORCID: https://orcid.org/0000-0002-9763-1794)
- Qin Zhang (ORCID: https://orcid.org/0000-0002-3981-0516)
- Hongjun Miao (ORCID: https://orcid.org/0000-0002-0739-3906)
Institutions
- Second Affiliated Hospital of Nanjing Medical University (CN)
- Nanjing Medical University (CN)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-02-26
- DOI
- https://doi.org/10.1186/s13023-025-03603-9
- Citations
- 4
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 6.50