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心肌病的临床与分子层面:新兴疗法与临床试验

Clinical and Molecular Aspects of Cardiomyopathies: Emerging Therapies and Clinical Trials.

作者信息

Maurizi Niccolò, Ammirati Enrico, Coppini Raffaele, Morrone Amelia, Olivotto Iacopo

机构信息

Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy.

"De Gasperis" Cardio Center and Transplant Center, ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy.

出版信息

Heart Fail Clin. 2018 Apr;14(2):161-178. doi: 10.1016/j.hfc.2018.01.001.

Abstract

Cardiomyopathies are diseases of the myocardium, often genetically determined, associated with heterogeneous phenotypes and clinical manifestations. Despite significant progress in the understanding of these conditions, available treatments mostly target late complications, whereas approaches that promise to interfere with the primary mechanisms and natural history are just beginning to surface. The last decade has witnessed the establishment of large international cardiomyopathy registries, paralleled by advances in cardiac imaging and genetic testing, deeper understanding of the pathophysiology and growing involvement by the pharmaceutical industry. As a result, the number of molecular interventions under scrutiny is increasing sharply.

摘要

心肌病是心肌疾病,通常由基因决定,伴有异质性表型和临床表现。尽管在对这些病症的认识上取得了重大进展,但现有治疗大多针对晚期并发症,而有望干预主要发病机制和自然病程的方法才刚刚开始出现。在过去十年中,大型国际心肌病注册机构得以建立,同时心脏成像和基因检测取得进展,对病理生理学有了更深入的了解,制药行业的参与度也不断提高。因此,正在接受审查的分子干预措施数量急剧增加。

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