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Cipaglucosidase alfa and miglustat for treatment of late-onset Pompe disease (LOPD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).

作者信息

Baker Elizabeth K, Derry Allyson, Cohen Jennifer L, Chang Irene J

机构信息

Division of Medical Genetics, Department of Pediatrics, Atrium Health Levine Children's Hospital, Charlotte, NC.

Lewis Katz School of Medicine at Temple University, Philadelphia, PA.

出版信息

Genet Med Open. 2025 Aug 25;3:103444. doi: 10.1016/j.gimo.2025.103444. eCollection 2025.

DOI:10.1016/j.gimo.2025.103444
PMID:40893835
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12398902/
Abstract
摘要

相似文献

1
Cipaglucosidase alfa and miglustat for treatment of late-onset Pompe disease (LOPD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).阿法糖苷酶和米格鲁司他治疗晚发型庞贝病（LOPD）：美国医学遗传学与基因组学学会（ACMG）的治疗公告
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2
Enzyme replacement therapy for late-onset Pompe disease.用于迟发性庞贝病的酶替代疗法。
Cochrane Database Syst Rev. 2023 Dec 12;12(12):CD012993. doi: 10.1002/14651858.CD012993.pub2.
3
An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease.阿伐糖苷酶α与西帕糖苷酶α加米格鲁司他治疗晚发型庞贝病患者的间接治疗比较
Adv Ther. 2025 Sep 8. doi: 10.1007/s12325-025-03301-9.
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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.西加葡萄糖苷酶 α 联合米格列醇与阿加糖酶 α 联合安慰剂治疗晚发性庞贝病（PROPEL）的安全性和有效性：一项国际性、随机、双盲、平行分组、3 期临床试验。
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Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL.将晚发性庞贝病的酶替代疗法从阿糖苷酶α转换为西糖苷酶α加米格鲁司他：PROPEL研究的事后效应量分析
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Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data.对比西加葡萄糖苷酶 α 联合米格列醇与其他酶替代疗法治疗晚发性庞贝病的疗效：利用患者水平和汇总数据进行的网络荟萃分析。
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本文引用的文献

1
Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.阿法糖苷酶α联合米格鲁司他：将作用机制与晚发型庞贝病的临床结局联系起来
Front Neurol. 2024 Oct 18;15:1451512. doi: 10.3389/fneur.2024.1451512. eCollection 2024.
2
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).阿法糖苷酶α联合米格鲁司他治疗晚发型庞贝病成人患者的104周疗效和安全性：一项III期开放标签扩展研究（ATB200-07）
J Neurol. 2024 May;271(5):2810-2823. doi: 10.1007/s00415-024-12236-0. Epub 2024 Feb 28.
3
Real-world outcomes from a series of patients with late onset Pompe disease who switched from alglucosidase alfa to avalglucosidase alfa.一系列晚发型庞贝病患者从阿糖苷酶α转换为阿伐糖苷酶α后的真实世界结果。
Front Genet. 2024 Jan 19;15:1309146. doi: 10.3389/fgene.2024.1309146. eCollection 2024.
4
Cipaglucosidase Alfa: First Approval.西格列葡萄糖苷酶：首次批准。
Drugs. 2023 Jun;83(8):739-745. doi: 10.1007/s40265-023-01886-5.
5
Early clinical phenotype of late onset Pompe disease: Lessons learned from newborn screening.晚发型庞贝病的早期临床表型：新生儿筛查的经验教训
Mol Genet Metab. 2022 Mar;135(3):179-185. doi: 10.1016/j.ymgme.2022.01.003. Epub 2022 Jan 23.
6
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.西加葡萄糖苷酶 α 联合米格列醇与阿加糖酶 α 联合安慰剂治疗晚发性庞贝病（PROPEL）的安全性和有效性：一项国际性、随机、双盲、平行分组、3 期临床试验。
Lancet Neurol. 2021 Dec;20(12):1027-1037. doi: 10.1016/S1474-4422(21)00331-8.
7
STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa.STIG研究：接受阿糖苷酶α酶替代疗法的庞贝病成人患者长期预后的真实世界数据。
J Neurol. 2021 Jul;268(7):2482-2492. doi: 10.1007/s00415-021-10409-9. Epub 2021 Feb 5.
8
Management of Confirmed Newborn-Screened Patients With Pompe Disease Across the Disease Spectrum.各种疾病谱中确诊新生儿筛查患者庞贝病的管理。
Pediatrics. 2017 Jul;140(Suppl 1):S24-S45. doi: 10.1542/peds.2016-0280E.
9
Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.晚期庞贝病患者接受阿糖苷酶 α 治疗后的生存和长期结局：系统评价和荟萃分析。
J Neurol. 2017 Apr;264(4):621-630. doi: 10.1007/s00415-016-8219-8. Epub 2016 Jul 2.
10
Consensus treatment recommendations for late-onset Pompe disease.迟发性庞贝病的共识治疗建议。
Muscle Nerve. 2012 Mar;45(3):319-33. doi: 10.1002/mus.22329. Epub 2011 Dec 15.