Onasemnogene abeparvovec - xii:世界上最昂贵和批准的治疗脊髓性肌萎缩症的药物

A. Pathan, V. Bairagi
{"title":"Onasemnogene abeparvovec - xii:世界上最昂贵和批准的治疗脊髓性肌萎缩症的药物","authors":"A. Pathan, V. Bairagi","doi":"10.37881/1.612","DOIUrl":null,"url":null,"abstract":"On May 24, 2019, the U.S. Food and Drug Administration approved Onasemnogene Abeparvovec-xioi (Zolgensma), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality. It is priced in the United States at $2.1m (£1.6m; €1.9m) the world’s most expensive drug. Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure. Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival. This review summarized the clinical guidelines for the Onasemnogene Abeparvovec-xioi use and may be useful to healthcare professionals.","PeriodicalId":383954,"journal":{"name":"NeuroPharmac Journal","volume":"1 1","pages":"0"},"PeriodicalIF":0.0000,"publicationDate":"2021-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Onasemnogene Abeparvovec-xioi: World’s most expensive and approved therapy for spinal muscular atrophy\",\"authors\":\"A. Pathan, V. Bairagi\",\"doi\":\"10.37881/1.612\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"On May 24, 2019, the U.S. Food and Drug Administration approved Onasemnogene Abeparvovec-xioi (Zolgensma), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality. It is priced in the United States at $2.1m (£1.6m; €1.9m) the world’s most expensive drug. Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure. Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival. This review summarized the clinical guidelines for the Onasemnogene Abeparvovec-xioi use and may be useful to healthcare professionals.\",\"PeriodicalId\":383954,\"journal\":{\"name\":\"NeuroPharmac Journal\",\"volume\":\"1 1\",\"pages\":\"0\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2021-04-30\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"NeuroPharmac Journal\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.37881/1.612\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"NeuroPharmac Journal","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.37881/1.612","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0

摘要

2019年5月24日,美国食品和药物管理局(fda)批准了Onasemnogene Abeparvovec-xioi (Zolgensma),这是首个被批准用于治疗两岁以下儿童脊髓性肌萎缩症(SMA)的基因疗法。脊髓性肌萎缩症是最严重的SMA,也是婴儿死亡的主要遗传原因。它在美国的售价为210万美元(160万英镑);190万欧元)是世界上最昂贵的药物。患有SMA的儿童在执行基本生活功能方面有困难。由于呼吸衰竭,大多数患有这种疾病的儿童无法存活过幼儿期。SMA患者现在有另一种治疗选择来最小化SMA的进展并提高生存率。这篇综述总结了Onasemnogene abparvovec - xii使用的临床指南,可能对医疗保健专业人员有用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Onasemnogene Abeparvovec-xioi: World’s most expensive and approved therapy for spinal muscular atrophy
On May 24, 2019, the U.S. Food and Drug Administration approved Onasemnogene Abeparvovec-xioi (Zolgensma), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality. It is priced in the United States at $2.1m (£1.6m; €1.9m) the world’s most expensive drug. Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure. Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival. This review summarized the clinical guidelines for the Onasemnogene Abeparvovec-xioi use and may be useful to healthcare professionals.
求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
自引率
0.00%
发文量
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信