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Nusinersen in spinal muscular atrophy type 1 from neonates to young adult: 1-year data from three Asia-Pacific regions.1型脊髓性肌萎缩症从新生儿到青年成人使用诺西那生的情况：来自亚太三个地区的1年数据
J Neurol Neurosurg Psychiatry. 2021 Nov;92(11):1244-1246. doi: 10.1136/jnnp-2020-324532. Epub 2021 Feb 11.
2
[Spinal muscular atrophy treated with nusinersen].[用诺西那生治疗脊髓性肌萎缩症]
Ugeskr Laeger. 2019 Jan 7;181(2).
3
Effect of Nusinersen on Respiratory Muscle Function in Different Subtypes of Type 1 Spinal Muscular Atrophy.诺西那生对1型脊髓性肌萎缩不同亚型呼吸肌功能的影响。
Am J Respir Crit Care Med. 2019 Dec 15;200(12):1547-1550. doi: 10.1164/rccm.201906-1175LE.
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Cost Effectiveness of Nusinersen in the Treatment of Patients with Infantile-Onset and Later-Onset Spinal Muscular Atrophy in Sweden.在瑞典，nusinersen 治疗婴儿期和晚发型脊髓性肌萎缩症患者的成本效益分析。
Pharmacoeconomics. 2019 Jun;37(6):845-865. doi: 10.1007/s40273-019-00769-6.
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Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study.Nusinersen 治疗成人 5q 型脊髓性肌萎缩症的非干预性、多中心、观察性队列研究。
Lancet Neurol. 2020 Apr;19(4):317-325. doi: 10.1016/S1474-4422(20)30037-5. Epub 2020 Mar 18.
6
Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges.实施婴儿型脊髓性肌萎缩症（I 型）全球扩展准入计划（EAP）：理解必要性、影响和挑战。
J Neuromuscul Dis. 2019;6(2):227-231. doi: 10.3233/JND-190387.
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Nusinersen for Type 1 Spinal Muscular Atrophy: A Father's Perspective.用于1型脊髓性肌萎缩症的诺西那生：一位父亲的视角
Pediatrics. 2019 Oct;144(4). doi: 10.1542/peds.2019-0226. Epub 2019 Sep 10.
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Resolution of skin necrosis after nusinersen treatment in an infant with spinal muscular atrophy.脊髓性肌萎缩症婴儿经诺西那生治疗后皮肤坏死的消退情况
Muscle Nerve. 2019 Jun;59(6):E42-E44. doi: 10.1002/mus.26457. Epub 2019 Mar 22.
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Stability of serial platelet and urine protein measurements in patients receiving nusinersen for spinal muscular atrophy.接受nusinersen 治疗的脊髓性肌萎缩症患者的血小板和尿蛋白连续测量的稳定性。
Muscle Nerve. 2022 Jul;66(1):76-79. doi: 10.1002/mus.27564. Epub 2022 May 11.
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Nusinersen improved respiratory function in spinal muscular atrophy type 2.诺西那生改善了2型脊髓性肌萎缩症患者的呼吸功能。
Pediatr Int. 2021 Aug;63(8):973-974. doi: 10.1111/ped.14510. Epub 2021 Jul 10.

引用本文的文献

1
Transforming Spinal Muscular Atrophy: From Pivotal Trials to Real-World Evidence and Future Therapeutic Frontiers in Types 1 and 2.转化型脊髓性肌萎缩症：从关键试验到1型和2型的真实世界证据及未来治疗前沿
Biomedicines. 2025 Aug 8;13(8):1939. doi: 10.3390/biomedicines13081939.
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Real-world analysis of the efficacy and safety of nusinersen in pediatric patients with spinal muscular atrophy.诺西那生钠治疗小儿脊髓性肌萎缩症疗效与安全性的真实世界分析
Orphanet J Rare Dis. 2025 Feb 26;20(1):87. doi: 10.1186/s13023-025-03603-9.
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Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis.基于基因的疗法治疗1型和2型脊髓性肌萎缩症：系统评价与荟萃分析
Gene Ther. 2024 Nov 27. doi: 10.1038/s41434-024-00503-8.
4
Analysis of spinal muscular atrophy carrier screening results in 32,416 pregnant women and 7,231 prepregnant women.对32416名孕妇和7231名孕前女性进行脊髓性肌萎缩症携带者筛查结果分析。
Front Neurol. 2024 Apr 9;15:1357476. doi: 10.3389/fneur.2024.1357476. eCollection 2024.
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Treatment of Symptomatic Spinal Muscular Atrophy with Nusinersen: A Prospective Longitudinal Study on Scoliosis Progression.用诺西那生治疗有症状的脊髓性肌萎缩症：关于脊柱侧弯进展的前瞻性纵向研究。
J Neuromuscul Dis. 2024;11(2):349-359. doi: 10.3233/JND-230077.
6
Nusinersen demonstrates effectiveness in treating spinal muscular atrophy: findings from a three-year nationwide study in Korea.诺西那生钠在治疗脊髓性肌萎缩症方面显示出有效性：韩国一项为期三年的全国性研究结果
Front Neurol. 2023 Dec 20;14:1294028. doi: 10.3389/fneur.2023.1294028. eCollection 2023.
7
Nusinersen for Spinal Muscular Atrophy Type I with Chronic Respiratory Failure: A Retrospective Study in South Korea.Ionis 反义寡核苷酸 nusinersen 治疗 1 型脊髓性肌萎缩伴慢性呼吸衰竭：韩国的一项回顾性研究。
Yonsei Med J. 2023 Dec;64(12):705-711. doi: 10.3349/ymj.2023.0080.
8
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.
9
New therapies for spinal muscular atrophy: where we stand and what is next.脊髓性肌萎缩症的新疗法：现状及未来展望。
Eur J Pediatr. 2023 Jul;182(7):2935-2942. doi: 10.1007/s00431-023-04883-8. Epub 2023 Apr 17.
10
Assessment of motor function and nutritional status in children with spinal muscular atrophy treated with nusinersen after loading period in Western China: a retrospective study.在中国西部，接受 nusinersen 负荷期治疗后的脊髓性肌萎缩症患儿的运动功能和营养状况评估：一项回顾性研究。
BMC Neurol. 2023 Jan 23;23(1):35. doi: 10.1186/s12883-023-03063-3.

本文引用的文献

1
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.在脊髓性肌萎缩症的前症状期对婴儿期开始用 nusinersen：2 期 NURTURE 研究的中期疗效和安全性结果。
Neuromuscul Disord. 2019 Nov;29(11):842-856. doi: 10.1016/j.nmd.2019.09.007. Epub 2019 Sep 12.
2
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy.SMA 类型 1 婴儿接受单次基因替换治疗的 1/2A 期研究中年龄和运动功能的影响。
Pediatr Neurol. 2019 Sep;98:39-45. doi: 10.1016/j.pediatrneurol.2019.05.005. Epub 2019 May 13.
3
Natural history of infantile-onset spinal muscular atrophy.婴儿型脊髓性肌萎缩症的自然病史。
Ann Neurol. 2017 Dec;82(6):883-891. doi: 10.1002/ana.25101. Epub 2017 Dec 8.
4
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.依库珠单抗治疗婴儿型脊髓性肌萎缩症的疗效观察
N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752.
5
Presymptomatic Diagnosis of Spinal Muscular Atrophy Through Newborn Screening.通过新生儿筛查对脊髓性肌萎缩症进行症状前诊断。
J Pediatr. 2017 Nov;190:124-129.e1. doi: 10.1016/j.jpeds.2017.06.042. Epub 2017 Jul 12.

Nusinersen in spinal muscular atrophy type 1 from neonates to young adult: 1-year data from three Asia-Pacific regions.

作者信息

Chan Sophelia Hoi-Shan, Chae Jong-Hee, Chien Yin-Hsiu, Ko Tae-Sung, Lee Jee Hun, Lee Yun Jeong, Nam Sang Ook, Jong Yuh-Jyh

机构信息

Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, The University of Hong Kong, Hong Kong SAR, China

Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine, Seoul, The Republic of Korea.

出版信息

J Neurol Neurosurg Psychiatry. 2021 Nov;92(11):1244-1246. doi: 10.1136/jnnp-2020-324532. Epub 2021 Feb 11.

DOI:10.1136/jnnp-2020-324532
PMID:33574081
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8522455/
Abstract
摘要