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A Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients with Cardiomyopathy due to MYBPC3 Mutations

The objective of this study is to follow, characterize and evaluate the natural history of the disease course, burden of illness, risk factors, quality of life (QoL), and biomarkers associated with disease progression and treatments in pediatric patients with cardiomyopathy (CM) due to MYBPC3 pathogenic or likely pathogenic mutations.


Why this Research Matters

This is a natural history study.


Who can Participate

Child and Teen

Children with a type of cardiomyopathy caused by a specific genetic mutation (MYBPC3 mutation) may be included in this research study.


Study ID

Protocol Number: 21-4206

Meet the Team

Image of Principal Investigator

Stephanie Nakano, MD

Principal Investigator