Hope on the horizon: FDA approves eplontersen for hereditary transthyretin-mediated amyloidosis.
作者信息
Binte Anwar Insa, Furqan Ahmed Iqra, Mahmood Fariha, Haseeb Abdul, Abbasher Hussien Mohamed Ahmed Khabab
机构信息
Liaquat National Medical College, Karachi, Pakistan.
Jinnah Sindh Medical University, Karachi, Pakistan.
出版信息
Ann Med Surg (Lond). 2025 Jan 9;87(1):20-23. doi: 10.1097/MS9.0000000000002839. eCollection 2025 Jan.
Abstract
摘要
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本文引用的文献
1
Effect of Eplontersen on Cardiac Structure and Function in Patients With Hereditary Transthyretin Amyloidosis.依洛硫酸酯酶治疗遗传性转甲状腺素蛋白淀粉样变性心肌病患者的心脏结构和功能的影响。
J Card Fail. 2024 Aug;30(8):973-980. doi: 10.1016/j.cardfail.2023.11.016. Epub 2023 Dec 7.
2
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy.依洛硫酸酯酶纳治疗遗传性转甲状腺素蛋白淀粉样变性多发性神经病。
JAMA. 2023 Oct 17;330(15):1448-1458. doi: 10.1001/jama.2023.18688.
3
Vutrisiran: A Review in Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis.伏硫西汀：遗传性转甲状腺素蛋白介导的淀粉样变性多发性神经病的综述
Drugs. 2023 Oct;83(15):1425-1432. doi: 10.1007/s40265-023-01943-z. Epub 2023 Sep 20.
4
Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis.正在开发用于转甲状腺素淀粉样变性的反义寡核苷酸 eplontersen 的群体药代动力学/药效学建模。
Br J Clin Pharmacol. 2022 Dec;88(12):5389-5398. doi: 10.1111/bcp.15468. Epub 2022 Aug 7.
5
Canadian Guidelines for Hereditary Transthyretin Amyloidosis Polyneuropathy Management.加拿大遗传性转甲状腺素蛋白淀粉样变性多发性神经病管理指南。
Can J Neurol Sci. 2022 Jan;49(1):7-18. doi: 10.1017/cjn.2021.34. Epub 2021 Feb 26.
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Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data.配体偶联反义寡核苷酸治疗转甲状腺素蛋白淀粉样变性：临床前和 1 期数据。
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Expert Rev Clin Pharmacol. 2019 Aug;12(8):701-711. doi: 10.1080/17512433.2019.1635008. Epub 2019 Jul 3.
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