RNA干扰治疗转甲状腺素型心脏淀粉样变性的疗效和安全性：一项系统综述和荟萃分析。

IF 4.4 3区医学 Q1 MEDICINE, GENERAL & INTERNAL

European Journal of Clinical Investigation Pub Date : 2025-04-17 DOI:10.1111/eci.70049

Shehroze Tabassum, Farhan Naeem, Mohamed Saad Rakab, Abdul Mannan Khan Minhas, Ramesh Daggubati, M. Chadi Alraies

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引用次数: 0

摘要

RNAi疗法代表了atr - cm治疗的一个有希望的进步，提供了比安慰剂显著的益处。它显示了生存率、功能能力和生活质量的显著改善，同时显著降低了BNP水平和gls关键的疾病进展生物标志物。这些发现强调了RNAi治疗在改变疾病轨迹和改善患者预后方面的潜力。

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Efficacy and safety of RNA interference therapeutics in transthyretin cardiac amyloidosis: A systematic review and meta-analysis

RNAi therapeutics represent a promising advancement in the treatment of ATTR-CM, offering significant benefits over placebo. It demonstrated substantial improvements in survival, functional capacity, and quality of life, alongside a marked reduction in BNP levels and GLS—critical biomarkers of disease progression. These findings underscore the potential of RNAi therapy to alter the disease trajectory and improve patient outcomes.

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来源期刊

European Journal of Clinical Investigation 医学-医学：内科

CiteScore

9.50

自引率

3.60%

发文量

192

审稿时长

1 months

期刊介绍： EJCI considers any original contribution from the most sophisticated basic molecular sciences to applied clinical and translational research and evidence-based medicine across a broad range of subspecialties. The EJCI publishes reports of high-quality research that pertain to the genetic, molecular, cellular, or physiological basis of human biology and disease, as well as research that addresses prevalence, diagnosis, course, treatment, and prevention of disease. We are primarily interested in studies directly pertinent to humans, but submission of robust in vitro and animal work is also encouraged. Interdisciplinary work and research using innovative methods and combinations of laboratory, clinical, and epidemiological methodologies and techniques is of great interest to the journal. Several categories of manuscripts (for detailed description see below) are considered: editorials, original articles (also including randomized clinical trials, systematic reviews and meta-analyses), reviews (narrative reviews), opinion articles (including debates, perspectives and commentaries); and letters to the Editor.