Long-Term Efficacy and Safety of ARRY-371797 (PF-0765803) in an Open-Label Rollover Study in Patients With Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation

Daniel P. Judge,Neal K. Lakdawala,Matthew R. Taylor,Luisa Mestroni,Huihua Li, Colleen Oliver,Franca S. Angeli,Patrice A. Lee,Calum Macrae

Circulation（2021）

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摘要

Introduction: Dilated cardiomyopathy due to LMNA gene mutations (LMNA-DCM) accounts for ~6% of DCM cases and is a life-threatening condition with a high unmet need. No disease-specific treatment ta...

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