Olivier Lairez , Pauline Fournier , Romain Itier , Bérengère Bachelet , Antoine Huart , Eve Cariou
{"title":"Towards etiological treatments in cardiomyopathies","authors":"Olivier Lairez , Pauline Fournier , Romain Itier , Bérengère Bachelet , Antoine Huart , Eve Cariou","doi":"10.1016/j.lpm.2024.104223","DOIUrl":null,"url":null,"abstract":"<div><p>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.</p></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"53 1","pages":"Article 104223"},"PeriodicalIF":3.2000,"publicationDate":"2024-02-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0755498224000010/pdfft?md5=6d8b5a4c246ae1a0c68755ee9e493237&pid=1-s2.0-S0755498224000010-main.pdf","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Presse Medicale","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0755498224000010","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, GENERAL & INTERNAL","Score":null,"Total":0}
引用次数: 0
Abstract
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.
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.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
