The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.
Study Type
OBSERVATIONAL
Enrollment
200
To characterize the disease course and natural history in participants with pathogenic or likely pathogenic MYBPC3 mutations with a specific focus on cardiac events and measurement
Time frame: 5 years for prospective group, n/a for retrospective group
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Arkansas Children's Hospital
Little Rock, Arkansas, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University of California Davis Health
Sacramento, California, United States
Rady Children's Hospital - San Diego
San Diego, California, United States
University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, United States
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, United States
Joe DiMaggio Children's Hospital
Hollywood, Florida, United States
Children's Mercy Hospital Kansas
Kansas City, Missouri, United States
St. Louis Children's Hospital
St Louis, Missouri, United States
Cohen Children's Medical Center
Lake Success, New York, United States
...and 19 more locations