心肌病的病因治疗。

IF 3.2 3区 医学 Q1 MEDICINE, GENERAL & INTERNAL Presse Medicale Pub Date : 2024-02-02 DOI:10.1016/j.lpm.2024.104223
Olivier Lairez , Pauline Fournier , Romain Itier , Bérengère Bachelet , Antoine Huart , Eve Cariou
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引用次数: 0

摘要

随着诊断技术的进步,心肌病的治疗方法也从机理方法转变为表型方法和病因学方法,本综述拟从这一角度探讨心肌病治疗方法的演变。文章超越了射血分数方法,探讨了针对心肌病病理生理途径的新疗法,这些疗法或针对表型,或针对病因。文章详细介绍了 HCM 治疗方法的演变,最后介绍了最新的病因治疗方法,如治疗肉瘤型 HCM 的马伐康坦、治疗转甲状腺素心脏淀粉样变性的他法米迪以及治疗法布里病的米格司他。此外,还回顾了肌球蛋白刺激剂在 DCM 治疗中的应用,然后探讨了基因疗法的前景,提出了直接治疗罪魁祸首基因突变的方法。
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Towards etiological treatments in cardiomyopathies

This review proposes to look at the evolution of cardiomyopathy treatments in the light of advances in diagnostic techniques, which have enabled to move from a mechanistic to a phenotypic and then etiological approach. The article goes beyond the ejection fraction approach, and look at new therapies that target the pathophysiological pathways of cardiomyopathies, either by targeting the phenotype, or by targeting the etiology. The evolution of HCM treatments is detailed, culminating in the latest etiological treatments such as mavacamten in sarcomeric HCM, tafamidis in transthyretin cardiac amyloidosis and migalastat in Fabry disease. Myosin stimulators are reviewed in the treatment of DCM, before opening perspectives for gene therapy, which proposes direct treatment of the culprit mutation.

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来源期刊
Presse Medicale
Presse Medicale 医学-医学:内科
自引率
3.70%
发文量
40
审稿时长
43 days
期刊介绍: Seule revue médicale "généraliste" de haut niveau, La Presse Médicale est l''équivalent francophone des grandes revues anglosaxonnes de publication et de formation continue. A raison d''un numéro par mois, La Presse Médicale vous offre une double approche éditoriale : - des publications originales (articles originaux, revues systématiques, cas cliniques) soumises à double expertise, portant sur les avancées médicales les plus récentes ; - une partie orientée vers la FMC, vous propose une mise à jour permanente et de haut niveau de vos connaissances, sous forme de dossiers thématiques et de mises au point dans les principales spécialités médicales, pour vous aider à optimiser votre formation.
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