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.
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.
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.
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.
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.
Can J Neurol Sci. 2022 Jan;49(1):7-18. doi: 10.1017/cjn.2021.34. Epub 2021 Feb 26.
6
Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data.
ESC Heart Fail. 2021 Feb;8(1):652-661. doi: 10.1002/ehf2.13154. Epub 2020 Dec 7.
7
Diagnosis and Treatment of Hereditary Transthyretin Amyloidosis (hATTR) Polyneuropathy: Current Perspectives on Improving Patient Care.
Ther Clin Risk Manag. 2020 Feb 21;16:109-123. doi: 10.2147/TCRM.S219979. eCollection 2020.
8
Tafamidis: A First-in-Class Transthyretin Stabilizer for Transthyretin Amyloid Cardiomyopathy.
Ann Pharmacother. 2020 May;54(5):470-477. doi: 10.1177/1060028019888489. Epub 2019 Nov 18.
9
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Expert Rev Clin Pharmacol. 2019 Aug;12(8):701-711. doi: 10.1080/17512433.2019.1635008. Epub 2019 Jul 3.
10
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Expert Rev Clin Pharmacol. 2019 Feb;12(2):95-99. doi: 10.1080/17512433.2019.1567326. Epub 2019 Jan 18.
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