Comprehensive assessment model for patients with spinal muscular atrophy: proposal of tools for clinical practice and real-world studies
- PMID: 36820780
- PMCID: PMC10004283
- DOI: 10.1590/1806-9282.20221608
Comprehensive assessment model for patients with spinal muscular atrophy: proposal of tools for clinical practice and real-world studies
Conflict of interest statement
Conflicts of interest: the authors declare there is no conflicts of interest.
References
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- Finkel RS, Mercuri E, Meyer OH, Simonds AK, Schroth MK, Graham RJ, et al. Diagnosis and management of spinal muscular atrophy: Part 2: pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromuscul Disord. 2018;28(3):197–207. doi: 10.1016/j.nmd.2017.11.004. - DOI - PubMed
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- Erdos J, Wild C. Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: a systematic review of real-world study data. Eur J Paediatr Neurol. 2022;39:1–10. doi: 10.1016/j.ejpn.2022.04.006. - DOI - PubMed
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