Suppr超能文献

派司奴单抗和依多萨林治疗遗传性转甲状腺素蛋白淀粉样变性的效果和价值。

The Effectiveness and Value of Patisiran and Inotersen for Hereditary Transthyretin Amyloidosis.

机构信息

1 Institute for Clinical and Economic Review, Boston, Massachusetts.

2 Boston University School of Medicine, Boston, Massachusetts.

出版信息

J Manag Care Spec Pharm. 2019 Jan;25(1):10-15. doi: 10.18553/jmcp.2019.25.1.010.

Abstract

Funding for this summary was contributed by the Laura and John Arnold Foundation, Blue Shield of California, and California Health Care Foundation to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, AHIP, Anthem, Blue Shield of California, CVS Caremark, Express Scripts, Harvard Pilgrim Health Care, Cambia Health Solutions, United Healthcare, Kaiser Permanente, Premera Blue Cross, AstraZeneca, Genentech, GlaxoSmithKline, Johnson & Johnson, Merck, National Pharmaceutical Council, Prime Therapeutics, Sanofi, Spark Therapeutics, Health Care Service Corporation, Editas, Alnylam, Regeneron, Mallinkrodt, Biogen, HealthPartners, and Novartis. Mickle, Dreitlein, and Pearson are ICER employees. Lasser, Cipriano, and Hoch have nothing to disclose.

摘要

本摘要的资金由 Laura and John Arnold 基金会、加州蓝盾和加州医疗保健基金会提供给临床与经济评价研究所(ICER),这是一个独立的组织,负责评估医疗保健干预措施的价值证据。ICER 的年度政策峰会由 Aetna、AHIP、Anthem、加州蓝盾、CVS Caremark、Express Scripts、Harvard Pilgrim Health Care、Cambia Health Solutions、United Healthcare、Kaiser Permanente、Premera Blue Cross、AstraZeneca、Genentech、GlaxoSmithKline、Johnson & Johnson、Merck、National Pharmaceutical Council、Prime Therapeutics、Sanofi、Spark Therapeutics、Health Care Service Corporation、Editas、Alnylam、Regeneron、Mallinkrodt、Biogen、HealthPartners 和 Novartis 等公司的会费支持。Mickle、Dreitlein 和 Pearson 是 ICER 的员工。Lasser、Cipriano 和 Hoch 没有要披露的信息。

相似文献

1
The Effectiveness and Value of Patisiran and Inotersen for Hereditary Transthyretin Amyloidosis.
J Manag Care Spec Pharm. 2019 Jan;25(1):10-15. doi: 10.18553/jmcp.2019.25.1.010.
2
The Effectiveness and Value of Lanadelumab and C1 Esterase Inhibitors for Prophylaxis of Hereditary Angioedema Attacks.
J Manag Care Spec Pharm. 2019 Feb;25(2):143-148. doi: 10.18553/jmcp.2019.25.2.143.
3
The Effectiveness and Value of Treatments for Spinal Muscular Atrophy.
J Manag Care Spec Pharm. 2019 Dec;25(12):1300-1306. doi: 10.18553/jmcp.2019.25.12.1300.
5
The Effectiveness and Value of Esketamine for the Management of Treatment-Resistant Depression.
J Manag Care Spec Pharm. 2020 Jan;26(1):16-20. doi: 10.18553/jmcp.2020.26.1.16.
6
The Effectiveness and Value of Biologic Therapies for the Treatment of Uncontrolled Asthma.
J Manag Care Spec Pharm. 2019 May;25(5):510-514. doi: 10.18553/jmcp.2019.25.5.510.
7
Cost-Effectiveness of Targeted Pharmacotherapy for Moderate to Severe Plaque Psoriasis.
J Manag Care Spec Pharm. 2018 Dec;24(12):1210-1217. doi: 10.18553/jmcp.2018.24.12.1210.
8
The Effectiveness and Value of Deflazacort and Exon-Skipping Therapies for the Management of Duchenne Muscular Dystrophy.
J Manag Care Spec Pharm. 2020 Apr;26(4):361-366. doi: 10.18553/jmcp.2020.26.4.361.
9
The Effectiveness and Value of Oral Semaglutide for Type 2 Diabetes Mellitus.
J Manag Care Spec Pharm. 2020 Sep;26(9):1072-1076. doi: 10.18553/jmcp.2020.26.9.1072.

引用本文的文献

3
A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis.
Neurol Ther. 2020 Dec;9(2):301-315. doi: 10.1007/s40120-020-00208-1. Epub 2020 Aug 12.
5
Disease-modifying therapy for proteinopathies: Can the exception become the rule?
Prog Mol Biol Transl Sci. 2019;168:277-287. doi: 10.1016/bs.pmbts.2019.07.010. Epub 2019 Aug 7.
6
Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle.
Nucleic Acids Res. 2019 Jul 9;47(12):6029-6044. doi: 10.1093/nar/gkz354.

本文引用的文献

2
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.
N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793.
3
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.
N Engl J Med. 2018 Jul 5;379(1):11-21. doi: 10.1056/NEJMoa1716153.
5
6
Safety and efficacy of a TTR specific antisense oligonucleotide in patients with transthyretin amyloid cardiomyopathy.
Amyloid. 2017 Dec;24(4):219-225. doi: 10.1080/13506129.2017.1374946. Epub 2017 Sep 14.
7
Evolving landscape in the management of transthyretin amyloidosis.
Ann Med. 2015;47(8):625-38. doi: 10.3109/07853890.2015.1068949. Epub 2015 Nov 27.
8
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study.
Orphanet J Rare Dis. 2015 Sep 4;10:109. doi: 10.1186/s13023-015-0326-6.
9
The amyloidogenic V122I transthyretin variant in elderly black Americans.
N Engl J Med. 2015 Jan 1;372(1):21-9. doi: 10.1056/NEJMoa1404852.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验