Article
Characterization and natural history of patients with LMNA-related dilated cardiomyopathy in the phase 3 REALM-DCM trial.
ESC heart failure - 1 Dec 2024
Garcia-Pavia Pablo, Lakdawala Neal K, Sinagra Gianfranco, Ripoll-Vera Tomas, Afshar Kia, Priori Silvia G, Ware James S, Owens Anjali, Li Huihua, Angeli Franca S, Elliott Perry, MacRae Calum A, Judge Daniel P
Abstract excerpt
AIMS: LMNA-related dilated cardiomyopathy (DCM) is a rare disease with an incompletely defined phenotype. The phase 3 REALM-DCM trial evaluated a potential disease-modifying therapy for LMNA-related DCM but was terminated due to futility without safety concern. This study utilized pooled data from REALM-DCM to descriptively characterize the phenotype and progression of LMNA-related DCM in a contemporary cohort of...
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