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Could choosing risdiplam instead of nusinersen in the treatment of type 1 spinal muscular atrophy be a huge cost-minimization opportunity?在1型脊髓性肌萎缩症的治疗中选择利司扑兰而非诺西那生钠会是一个巨大的成本最小化机会吗？
Croat Med J. 2024 Oct 31;65(5):454-456. doi: 10.3325/cmj.2024.65.454.
2
Cost-Effectiveness of Technologies for the Treatment of Spinal Muscular Atrophy: A Systematic Review of Economic Studies.技术治疗脊髓性肌萎缩症的成本效益：经济研究的系统评价。
Value Health Reg Issues. 2024 Jul;42:100985. doi: 10.1016/j.vhri.2024.02.002. Epub 2024 Apr 26.
3
Cost-Effectiveness of Onasemnogene Abeparvovec Compared With Nusinersen and Risdiplam in Patients With Spinal Muscular Atrophy Type 1 in Brazil: Custo-Efetividade do Onasemnogeno Abeparvoveque (AVXS-101) em Comparação ao Nusinersena e Risdiplam em Pacientes com Atrofia Muscular Espinhal Tipo 1 no Brasil.巴西脊髓性肌萎缩症 1 型患者接受onasemnogene abeparvovec、nusinersen 和 risdiplam 的成本-效果比较：Custo-Efetividade do Onasemnogeno Abeparvoveque (AVXS-101) em Comparação ao Nusinersena e Risdiplam em Pacientes com Atrofia Muscular Espinhal Tipo 1 no Brasil.
Value Health Reg Issues. 2024 Mar;40:108-117. doi: 10.1016/j.vhri.2023.11.004. Epub 2024 Jan 5.
4
Long-Term Comparative Efficacy and Safety of Risdiplam and Nusinersen in Children with Type 1 Spinal Muscular Atrophy.利司扑兰与nusinersen 治疗 1 型脊髓性肌萎缩症儿童的长期疗效和安全性比较。
Adv Ther. 2024 Jun;41(6):2414-2434. doi: 10.1007/s12325-024-02845-6. Epub 2024 May 5.
5
A case series evaluating patient perceptions after switching from nusinersen to risdiplam for spinal muscular atrophy.评估患者在从依洛硫酸酯酶β（nusinersen）转换为利司扑兰（risdiplam）治疗脊髓性肌萎缩症后的认知改变的病例系列研究。
Muscle Nerve. 2024 Feb;69(2):179-184. doi: 10.1002/mus.28015. Epub 2023 Dec 1.
6
How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison.与其他治疗1-3型脊髓性肌萎缩症的方法相比，利司扑兰的效果如何：一项系统文献综述和间接治疗比较
J Comp Eff Res. 2022 Apr;11(5):347-370. doi: 10.2217/cer-2021-0216. Epub 2022 Jan 18.
7
Safety of risdiplam in spinal muscular atrophy patients after short-term treatment with nusinersen.利司扑兰治疗脊髓性肌萎缩症患者短期应用依洛硫酸酯酶β后的安全性。
Muscle Nerve. 2024 Nov;70(5):1095-1098. doi: 10.1002/mus.28228. Epub 2024 Aug 13.
8
Budget impact analysis: comparing the costs of nusinersen and risdiplam in the treatment of type 3 spinal muscular atrophy patients.
Eur J Hosp Pharm. 2025 Apr 23;32(3):293-294. doi: 10.1136/ejhpharm-2023-003733.
9
Safety and Efficacy of Nusinersen and Risdiplam for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials.诺西那生钠和利司扑兰治疗脊髓性肌萎缩症的安全性和有效性：一项随机对照试验的系统评价和荟萃分析
Brain Sci. 2023 Oct 7;13(10):1419. doi: 10.3390/brainsci13101419.
10
Switching from Nusinersen to Risdiplam: A Croatian Real-World Experience on Effectiveness and Safety.从诺西那生钠转换为利司扑兰：克罗地亚关于有效性和安全性的真实世界经验。
J Pers Med. 2024 Feb 24;14(3):244. doi: 10.3390/jpm14030244.

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1
Bridging Clinical Outcomes and Cost-Effectiveness: The Role of Real-World Data in Cystic Fibrosis Therapy.连接临床结果与成本效益：真实世界数据在囊性纤维化治疗中的作用。
Pharmacol Res Perspect. 2025 Aug;13(4):e70138. doi: 10.1002/prp2.70138.

本文引用的文献

1
Switching from Nusinersen to Risdiplam: A Croatian Real-World Experience on Effectiveness and Safety.从诺西那生钠转换为利司扑兰：克罗地亚关于有效性和安全性的真实世界经验。
J Pers Med. 2024 Feb 24;14(3):244. doi: 10.3390/jpm14030244.
2
Challenges and opportunities in spinal muscular atrophy therapeutics.脊髓性肌萎缩症治疗的挑战与机遇。
Lancet Neurol. 2024 Feb;23(2):205-218. doi: 10.1016/S1474-4422(23)00419-2.
3
A case series evaluating patient perceptions after switching from nusinersen to risdiplam for spinal muscular atrophy.评估患者在从依洛硫酸酯酶β（nusinersen）转换为利司扑兰（risdiplam）治疗脊髓性肌萎缩症后的认知改变的病例系列研究。
Muscle Nerve. 2024 Feb;69(2):179-184. doi: 10.1002/mus.28015. Epub 2023 Dec 1.
4
Safety Concerns with Nusinersen, Risdiplam, and Onasemnogene Abeparvovec in Spinal Muscular Atrophy: A Real-World Pharmacovigilance Study.脊髓性肌萎缩症中使用 nusinersen、risdiplam 和 onasemnogene abeparvovec 的安全性问题：一项真实世界的药物警戒研究。
Clin Drug Investig. 2023 Dec;43(12):949-962. doi: 10.1007/s40261-023-01320-4. Epub 2023 Nov 23.
5
Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.接受过其他脊髓性肌萎缩症治疗的患者使用利司扑兰的情况：来自JEWELFISH研究的中期分析
Neurol Ther. 2023 Apr;12(2):543-557. doi: 10.1007/s40120-023-00444-1. Epub 2023 Feb 13.
6
An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.利司扑兰用于 1 型或 2 型脊髓性肌萎缩症患者的扩展使用项目。
Ann Clin Transl Neurol. 2022 Jun;9(6):810-818. doi: 10.1002/acn3.51560. Epub 2022 May 14.
7
How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison.与其他治疗1-3型脊髓性肌萎缩症的方法相比，利司扑兰的效果如何：一项系统文献综述和间接治疗比较
J Comp Eff Res. 2022 Apr;11(5):347-370. doi: 10.2217/cer-2021-0216. Epub 2022 Jan 18.
8
Risdiplam: new opportunities but more to be done.利司扑兰：新机遇，但仍有更多工作要做。
Lancet Neurol. 2022 Jan;21(1):23-24. doi: 10.1016/S1474-4422(21)00428-2.
9
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.针对 1 型脊髓性肌萎缩症（SMA）的婴儿期起病症状性的 Onasemnogene abeparvovec 基因治疗（STR1VE-EU）：一项开放标签、单臂、多中心、3 期临床试验。
Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9.
10
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.Risdiplam 治疗 1 型脊髓性肌萎缩症婴儿与历史对照。
N Engl J Med. 2021 Jul 29;385(5):427-435. doi: 10.1056/NEJMoa2102047.

Could choosing risdiplam instead of nusinersen in the treatment of type 1 spinal muscular atrophy be a huge cost-minimization opportunity?

作者信息

Belančić Andrej, Faour Andrea Katrin, Gkrinia Elvira Meni Maria, Vitezić Dinko

机构信息

Andrej Belančić, Department of Clinical Pharmacology, Clinical Hospital Centre Rijeka, Rijeka, Croatia,

出版信息

Croat Med J. 2024 Oct 31;65(5):454-456. doi: 10.3325/cmj.2024.65.454.

DOI:10.3325/cmj.2024.65.454
PMID:39492456
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11568377/
Abstract
摘要