Mavacamten shows broad benefit in human and mouse models of MYBPC3-related hypertrophic cardiomyopathy.

doi:10.1038/s44161-026-00833-3

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Article

Mavacamten shows broad benefit in human and mouse models of MYBPC3-related hypertrophic cardiomyopathy.

Nature cardiovascular research - 1 Jul 2026

Sen-Martín Laura, Fernández-Trasancos Ángel, López-Unzu Miguel Á, Bak Agata, Zafra-Castellano Magdalena, Srikantharajah Rajiven, Pathak Divya, Klotz Annika, Kuehn Michel N, Ferrarini Alessia, Labrador-Cantarero Verónica, Sánchez-Ortiz David, Pricolo María Rosaria, Vicente Natalia, Velázquez-Carreras Diana, Sánchez-García Lucía, Sicilia Jon, Nicolás-Ávila José Ángel, Sánchez-Díaz María, Schlossarek Saskia, Cussó Lorena, Desco Manuel, Villalba-Orero María, Guzmán-Martínez Gabriela, Calvo Enrique, Barriales-Villa Roberto, Vázquez Jesús, Sánchez-Cabo Fátima, Hidalgo Andrés, de Tombe Pieter P, Carrier Lucie, Spudich James A, Ruppel Kathleen M, Weinberger Florian, Cazorla Olivier, Hessel Anthony L, Alegre-Cebollada Jorge

Abstract excerpt

Mavacamten is a targeted treatment for hypertrophic cardiomyopathy, a disease caused by genetic variants affecting mainly sarcomeric myosin and its regulator cardiac myosin-binding protein C (cMyBP-C, encoded by MYBPC3). Here we generate knock-in mice including missense pathogenic variant cMyBP-C p.R502W, which unlike carriers of cMyBP-C truncations, develop pathogenic myocardial remodeling with preserved cMyBP-C...

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Identifiers

PMID: 42414613
DOI: 10.1038/s44161-026-00833-3

Genes

MYBPC3

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

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