加强鞘内注射和基因治疗的临床基础设施:针对 SOD1-ALS 患者的 Qalsody (Tofersen) 模型。

IF 2.3 Q3 CLINICAL NEUROLOGY
Neurology. Clinical practice Pub Date : 2024-08-01 Epub Date: 2024-05-16 DOI:10.1212/CPJ.0000000000200303
Jennifer Morganroth, Tanya M Bardakjian, Laynie Dratch, Colin C Quinn, Lauren B Elman
{"title":"加强鞘内注射和基因治疗的临床基础设施:针对 SOD1-ALS 患者的 Qalsody (Tofersen) 模型。","authors":"Jennifer Morganroth, Tanya M Bardakjian, Laynie Dratch, Colin C Quinn, Lauren B Elman","doi":"10.1212/CPJ.0000000000200303","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Qalsody (tofersen), an intrathecal therapy (IT) antisense oligonucleotide (ASO), was granted accelerated approval by the Food and Drug Administration for the treatment of <i>SOD1</i>-mediated amyotrophic lateral sclerosis (ALS) on April 25, 2023. Academic centers need to be prepared for expedited drug delivery. The purpose of this model was to predict the number of <i>SOD1</i>-ALS patients whom we expect to see at our center at the time of Qalsody approval and to use it to extrapolate to a model for a hypothetical sporadic IT ALS therapy.</p><p><strong>Recent findings: </strong>We predicted that 6 symptomatic and 14 presymptomatic <i>SOD1</i> patients would come to our center, whereas a sporadic therapy would generate 108 patients, creating excess office visits, lumbar punctures, and genetic counseling visits.</p><p><strong>Implications for practice: </strong>As new therapies for neurologic diseases come to market, preparing for increased office volume and complex drug delivery are essential for optimal care.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":null,"pages":null},"PeriodicalIF":2.3000,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11157423/pdf/","citationCount":"0","resultStr":"{\"title\":\"Enhancing Clinical Infrastructure for the Delivery of Intrathecal and Genetic Therapies: A Qalsody (Tofersen) Model for Patients With <i>SOD1</i>-ALS.\",\"authors\":\"Jennifer Morganroth, Tanya M Bardakjian, Laynie Dratch, Colin C Quinn, Lauren B Elman\",\"doi\":\"10.1212/CPJ.0000000000200303\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Qalsody (tofersen), an intrathecal therapy (IT) antisense oligonucleotide (ASO), was granted accelerated approval by the Food and Drug Administration for the treatment of <i>SOD1</i>-mediated amyotrophic lateral sclerosis (ALS) on April 25, 2023. Academic centers need to be prepared for expedited drug delivery. The purpose of this model was to predict the number of <i>SOD1</i>-ALS patients whom we expect to see at our center at the time of Qalsody approval and to use it to extrapolate to a model for a hypothetical sporadic IT ALS therapy.</p><p><strong>Recent findings: </strong>We predicted that 6 symptomatic and 14 presymptomatic <i>SOD1</i> patients would come to our center, whereas a sporadic therapy would generate 108 patients, creating excess office visits, lumbar punctures, and genetic counseling visits.</p><p><strong>Implications for practice: </strong>As new therapies for neurologic diseases come to market, preparing for increased office volume and complex drug delivery are essential for optimal care.</p>\",\"PeriodicalId\":19136,\"journal\":{\"name\":\"Neurology. Clinical practice\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2024-08-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11157423/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Neurology. Clinical practice\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1212/CPJ.0000000000200303\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/5/16 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Neurology. Clinical practice","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1212/CPJ.0000000000200303","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/5/16 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
引用次数: 0

摘要

背景:美国食品和药物管理局于 2023 年 4 月 25 日加速批准 Qalsody(托福生)作为一种鞘内治疗(IT)反义寡核苷酸(ASO)用于治疗 SOD1 介导的肌萎缩性脊髓侧索硬化症(ALS)。学术中心需要为加速给药做好准备。该模型的目的是预测在 Qalsody 获批时我们预计在本中心就诊的 SOD1-ALS 患者人数,并以此推断假设的散发性 IT ALS 治疗模型:我们预测,6 名有症状的 SOD1 患者和 14 名无症状的 SOD1 患者将到我们中心就诊,而散发性疗法将产生 108 名患者,从而产生过多的门诊、腰椎穿刺和遗传咨询门诊:随着神经系统疾病新疗法的上市,为增加诊疗量和复杂的给药方式做好准备对于优化医疗服务至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Enhancing Clinical Infrastructure for the Delivery of Intrathecal and Genetic Therapies: A Qalsody (Tofersen) Model for Patients With SOD1-ALS.

Background: Qalsody (tofersen), an intrathecal therapy (IT) antisense oligonucleotide (ASO), was granted accelerated approval by the Food and Drug Administration for the treatment of SOD1-mediated amyotrophic lateral sclerosis (ALS) on April 25, 2023. Academic centers need to be prepared for expedited drug delivery. The purpose of this model was to predict the number of SOD1-ALS patients whom we expect to see at our center at the time of Qalsody approval and to use it to extrapolate to a model for a hypothetical sporadic IT ALS therapy.

Recent findings: We predicted that 6 symptomatic and 14 presymptomatic SOD1 patients would come to our center, whereas a sporadic therapy would generate 108 patients, creating excess office visits, lumbar punctures, and genetic counseling visits.

Implications for practice: As new therapies for neurologic diseases come to market, preparing for increased office volume and complex drug delivery are essential for optimal care.

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
Neurology. Clinical practice
Neurology. Clinical practice CLINICAL NEUROLOGY-
CiteScore
4.00
自引率
0.00%
发文量
77
期刊介绍: Neurology® Genetics is an online open access journal publishing peer-reviewed reports in the field of neurogenetics. The journal publishes original articles in all areas of neurogenetics including rare and common genetic variations, genotype-phenotype correlations, outlier phenotypes as a result of mutations in known disease genes, and genetic variations with a putative link to diseases. Articles include studies reporting on genetic disease risk, pharmacogenomics, and results of gene-based clinical trials (viral, ASO, etc.). Genetically engineered model systems are not a primary focus of Neurology® Genetics, but studies using model systems for treatment trials, including well-powered studies reporting negative results, are welcome.
×
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