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.
Abstract
摘要
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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？
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本文引用的文献
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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.
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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
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Ann Clin Transl Neurol. 2022 Jun;9(6):810-818. doi: 10.1002/acn3.51560. Epub 2022 May 14.
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J Comp Eff Res. 2022 Apr;11(5):347-370. doi: 10.2217/cer-2021-0216. Epub 2022 Jan 18.
8
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Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9.
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N Engl J Med. 2021 Jul 29;385(5):427-435. doi: 10.1056/NEJMoa2102047.
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