TTR 基因沉默剂治疗遗传性ATTR 淀粉样变性：不仅仅是 ICER 认可的。
TTR Gene Silencers for Hereditary ATTR Amyloidosis: More than ICER Recognized.
机构信息
1 Assistant Director, Amyloidosis Center, Boston University Medical Center, Boston, Massachusetts.
出版信息
J Manag Care Spec Pharm. 2019 Jan;25(1):15-16. doi: 10.18553/jmcp.2019.25.1.015.
Abstract
No funding support was received for the writing of this commentary. The author ser ves as a consultant for Akcea Therapeutics, Alnylam Phar maceuticals, Corino Therapeutics, Intellia Therapeutics, and Ionis Pharmaceuticals.
摘要
本文评论撰写没有获得任何资金支持。作者担任 Akcea Therapeutics、Alnylam Phar maceuticals、Corino Therapeutics、Intellia Therapeutics 和 Ionis Pharmaceuticals 的顾问。
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本文引用的文献
1
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.
N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793.
2
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.
N Engl J Med. 2018 Jul 5;379(1):11-21. doi: 10.1056/NEJMoa1716153.
3
Oligonucleotide Drugs for Transthyretin Amyloidosis.
N Engl J Med. 2018 Jul 5;379(1):82-85. doi: 10.1056/NEJMe1805499.
4
Enzalutamide in Men with Nonmetastatic, Castration-Resistant Prostate Cancer.
N Engl J Med. 2018 Jun 28;378(26):2465-2474. doi: 10.1056/NEJMoa1800536.
5
Apalutamide Treatment and Metastasis-free Survival in Prostate Cancer.
N Engl J Med. 2018 Apr 12;378(15):1408-1418. doi: 10.1056/NEJMoa1715546. Epub 2018 Feb 8.
6
Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy.
BMC Neurol. 2017 Sep 11;17(1):181. doi: 10.1186/s12883-017-0948-5.
7
Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.
JAMA. 2013 Dec 25;310(24):2658-67. doi: 10.1001/jama.2013.283815.
8
Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial.
Neurology. 2012 Aug 21;79(8):785-92. doi: 10.1212/WNL.0b013e3182661eb1. Epub 2012 Jul 25.
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